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Quarterly Drug Pipeline: January 2025

Clinical insights and competitive intelligence on anticipated drugs in development

January 30, 2025

Editor-in-chief's message

Welcome to the Prime Quarterly Drug Pipeline! Dive into clinical insights and competitive intelligence on anticipated drugs in development, so you are well-sourced on the drug pipeline.

Methodology

The drug pipeline is complex and fluid. Our talented and committed team of clinical and analytics experts are excited to bring you this robust publication after thoughtful research. Specialty and traditional drugs that are covered under the pharmacy and medical benefits are featured. New molecular entities, pertinent new and expanded indications for existing medications, biosimilars, and regenerative medicines, such as gene and cellular therapies, are also profiled.

Quarterly Drug Pipeline details both agents submitted for FDA review and those in phase 3 studies with a likelihood to apply to the FDA. Our Deep Dives consider the evidence, the products’ potential to fill an unmet need or become the new standard of care, and the ability to replace existing therapies.

A market agnostic financial forecast primarily from Evaluate is included for select agents to assist payers with assessing the potential budgetary impact of the pipeline. Five-year projected annual U.S. sales are forecasted.

Reflection
In 2024, the FDA approved 50 novel drugs. While this is slightly lower than the year prior – 55 novel drugs were approved in 2023 – it is higher than the FDA’s 10-year novel drug approval average of about 47 per year. More than half of novel approvals in 2024 were for rare or orphan diseases. Moreover, 66% of novel approvals underwent at least one of the Agency’s expedited programs to speed approval for serious conditions, with 16% as Accelerated Approval. This designation can be challenging for payors to manage, since approval is based on a surrogate endpoint thought to predict clinical benefit. While numbers do not tell the entire story, they represent innovation for patient care and have the potential to advance health for the American public.

On the horizon
The FDA decisions for specialty medications (71%) and for Orphan Drugs (36%) continue to grow for agents with applications submitted to the FDA. Nine agents are seeking FDA’s Accelerated Approval.

First quarter 2025 may usher in notable approvals. Select highlights include:

A new mechanism for select patients with Duchenne muscular dystrophy (DMD)
First encapsulated cell therapy as an intravitreal implant for macular telangiectasia type 2, a macular degenerative disease
First-in-class therapeutic for uncomplicated urinary tract infections
SC therapeutic for hemophilia A and B (with and without inhibitors)
A self-administered intranasal formulation for paroxysmal supraventricular tachycardia (PSVT)
New options for unresectable liver cancer
First agent for Prader-Willi syndrome (PWS) a rare genetic disorder with a common syndromic manifestation of obesity
An expanded indication for intranasal epinephrine for anaphylaxis to include pediatric patients with lower weight

We hope you enjoy the report!

Maryam Tabatabai
Associate Vice President, Clinical Information

Editorial team

Maryam Tabatabai, PharmD

Editor-In-Chief
Associate Vice President, Clinical Information

Carole Kerzic, RPh
Executive Editor
Drug Information Pharmacist Principal

Nicole Kjesbo, PharmD, BCPS
Executive Editor
Clinical Program Development Director Senior, Pipeline

Consultant panel

Robert Greer, RPh, BCOP
Vice President, Clinical Strategy and Programs

Andrea Henry, PharmD, MBA, BCPS
Drug Information Pharmacist Principal

Danny Melson
Data Scientist Principal

Olivia Pane, PharmD, CDCES
Drug Information Pharmacist

Natalee Felten, PharmD, BCPS
Medical Pharmacy Clinical Pharmacist

All brand names are property of their respective owners.

The drug pipeline is fluid; the dates and information within this publication are subject to change. Nothing herein is or shall be construed as a promise or representation regarding past or future events and Prime Therapeutics expressly disclaims any and all liability relating to the use of or reliance on the information contained in this presentation. The information contained in this publication is intended for educational purposes only and should not be considered clinical, financial, or legal advice. By receipt of this publication, each recipient agrees that the information contained herein will be kept confidential and that the information will not be photocopied, reproduced, distributed to, or disclosed to others at any time without the prior written consent of Prime Therapeutics.

Deep dive

The Deep Dive section features an analysis for select pipeline drugs that are expected to be FDA-approved in the upcoming quarters. Selected agents are expected to have a high clinical and/or financial impact on healthcare. Agent selection considers the evidence — the products’ potential to fill an unmet need, become the new standard of care, or the ability to replace existing therapies. Typically, Deep Dives focus on new moieties, however, existing drugs that may offer a novel mechanism of action for existing conditions may be featured. Agents granted designations from the FDA to expedite review and/or support development and evaluation of pipeline drugs are also considered.

Specialty drug names appear in red throughout the publication.

ataluren (Translarna) oral

Manufacturer: PTC Therapeutics

Proposed indications

Nonsense mutation Duchenne muscular dystrophy (nmDMD)

Clinical overview

Mechanism of action

Ataluren, a protein restoration therapy, allows the cellular process that synthesizes dystrophin protein to bypass the genetic defect (nonsense mutation) in the dystrophin gene, thereby producing a functional dystrophin protein.

Clinical trials

Ataluren was evaluated in an international, double-blind, placebo-controlled, Phase 3 trial (Study 041; NCT03179631) in 359 boys ≥ 5 years of age with documented nmDMD. Enrolled patients were on a stable corticosteroid regimen and displayed a 6MWD ≥ 150 meters. In the primary analysis population, ataluren did not reach statistical significance in the primary endpoint of mean 6MWD change from baseline (difference from placebo, 8.3 meters; p=0.3626) at 72 weeks. However, ataluren did lead to a significant difference in the mean 6MWD change from baseline compared to placebo in the ITT population (difference from placebo, 14.4 meters; p=0.0248), which included boys who received at least one dose of study treatment; this translated to a 21% slowing of the rate of decline in 6MWD in the ITT population. The greatest benefit, reported as a 30% slowing in rate of decline in 6MWD, was observed among patients with 6MWD of 300 to 400 meters at baseline (difference in change compared to placebo, 24.2 meters; p=0.031). In addition, significant benefit was proved with ataluren compared to placebo in key secondary endpoints, including the NorthStar Ambulatory Assessment (p=0.0283), 10-meter walk/run (p=0.0422), 4-stair climb (p=0.0293), and time to 10% worsening of 6MWD (p=0.0078). Ataluren was well-tolerated.

The long-term, international, observational STRIDE study (NCT02369731) has provided long-term real-world data of ataluren in routine care. The effectiveness of ataluren is assessed by comparing results from propensity score-matched patient populations from the STRIDE registry, including patients with nmDMD treated with ataluren and SOC, with data from the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) that included patients with any DMD genotype receiving SOC alone (control group). The study revealed ataluren led to a 3.5-year delay in loss of ambulation (p<0.0001) and a 1.8-year delay in reaching a predicted FVC of < 60% (p=0.0028), an important threshold of lung function.

Notably, the prior double-blind, placebo-controlled, Phase 3 ACT DMD trial (NCT01826487; n=228 ITT) in men 7–16 years of age with nmDMD reported that ataluren did not lead to a significant change from baseline in the primary endpoint of 6MWD compared to placebo (difference compared to placebo, 13 meters; p=0.213) at week 48; however, a significant difference (15.9 meters) in change in 6MWD and a slower rate of decline in physical function were seen between the ataluren and placebo groups in a prespecified subgroup of patients who had a baseline 6MWD of 300 meters to < 400 meters.

Dosage and administration

In the clinical trials, ataluren was administered orally as 40 mg/kg/day in three divided doses (10 mg/kg, 10 mg/kg, 20 mg/kg).

Place in therapy

DMD is a rare, X-linked neuromuscular disorder characterized by progressive muscle degeneration and weakness. It is estimated that 400 to 600 boys are born with DMD each year in the U.S. In DMD, dystophin gene mutations lead to a lack of functional dystrophin, a protein involved in maintaining muscle fiber integrity. Approximately 13% of cases (~2,000 cases) of DMD in the U.S. have nonsense mutations. A nonsense mutation is an alteration in the genetic code that prematurely stops the synthesis of a protein, resulting in an incomplete, dysfunctional protein. Onset of signs and symptoms of DMD occurs between 2–5 years of age. Most boys affected lose the ability to walk by 12 years of age. Moreover, death due to respiratory or cardiac failure typically occurs by the early 30s.

Corticosteroids may delay progression of muscle weakness and improve respiratory function and have been the SOC for patients with DMD. Deflazacort (Emflaza) and vamorolone (Agamree) are oral corticosteroids that are FDA-approved specifically for DMD; prednisone has also been prescribed off-label for DMD. Corticosteroids may be used in combination with other agents for DMD, including the IV administered antisense oligonucleotides casimersen (Amondys 45), eteplirsen (Exondys 51), golodirsen (Vyondys 53) and viltolarsen (Viltepso), and the oral histone deacetylase inhibitor givinostat (Duvyzat). Sarepta’s delandistrogene moxeparvovec-rokl (Elevidys) is the only gene therapy approved to treat DMD.

Ataluren has met challenges in seeking FDA approval for DMD. Some trials did not meet the primary efficacy endpoint (6MWD) leading to the FDA’s advisory committee vote that the data were inconclusive to establish ataluren’s effectiveness in DMD and the FDA to issue complete response letters. Study 041 was submitted to satisfy the data requirement for resubmission and proved a significant slowing of the rate of decline in ambulation (6MWD) after 72 weeks of therapy with ataluren, particularly among patients with a baseline 6MWD of 300 to < 400 meters. If approved, ataluren will offer a new mechanism of action for DMD by circumventing the dystrophin gene errors to produce a functional dystrophin protein. Its use is expected to be limited to patients with nonsense mutations in the dystrophin gene.

FDA approval timeline

January to March 2025

FDA designations: Fast Track, Orphan Drug

Financial forecast (reported in millions)

The financial forecast for ataluren is not currently available.

avutometinib + defactinib oral

Manufacturer: Verastem / Pfizer

Proposed indications

Recurrent KRAS mutant low-grade serous ovarian cancer (LGSOC) after at least one prior systemic therapy

Clinical overview

Mechanism of action

Avutometinib is a RAF/MEK clamp agent. It inhibits MEK signaling without the compensatory activation of MEK, which could limit the efficacy of MEK-only inhibitors.

Defactinib is a selective focal adhesion kinase (FAK) inhibitor that inhibits parallel pathway signaling and has demonstrated synergy with avutometinib.

Clinical trial(s)

The randomized, open-label, Phase 2 RAMP-201 trial (NCT04625270) evaluated avutometinib in combination with defactinib in patients with recurrent LGSOC who received prior treatment with chemotherapy and/or MEK inhibitor therapy and/or bevacizumab. Mature data (cut off June 30, 2024) from the Part B expansion phase of the trial revealed the primary endpoint of ORR was 44% among patients with KRAS mutated LGSOC with measurable disease and approximately 12 months of follow-up. Secondary outcomes among this patient population included a median DOR of 31.1 months, median PFS of 22 months, and DCR at least six months of 70%. Notably, the trial reported lower response rates and duration of effect in enrolled patients with KRAS wild-type LGSOC (ORR, 17%; median DOR, 9.2 months; median PFS, 12.8 months; DCR, 50%). The most common TEAEs (all grades, grade ≥3) for the combination therapy were nausea (67%, 2.6%), diarrhea (58.3%, 7.8%), and increased blood creatine phosphokinase levels (60%, 24.3%). Ten percent of patients discontinued treatment due to adverse events.

The FDA submission was also supported by data from the Phase 1, FRAME study (NCT03875820) that demonstrated an ORR of 58% among patients with KRAS mutant LGSOC (n=12) who were treated with avutometinib plus defactinib.

Dosage and administration

Patients received avutometinib 3.2 mg orally twice weekly and defactinib 200 mg orally twice daily during Part B of the RAMP 201 study. Both agents were administered for three weeks followed by one week off in every four-week cycle.

Place in therapy

Ovarian cancer is the second most common gynecologic cancer in the U.S. It is estimated that about 19,680 new cases of ovarian cancer were diagnosed in 2024 and approximately 12,740 deaths occurred from the disease. LGSOC accounts for approximately 5% of ovarian cancer cases. LGSOC typically affects women at a relatively young age (average, 43 years). While it usually develops more slowly than other types of ovarian carcinomas, it is often diagnosed at advanced stages.

The RAS/RAF/MEK/ERK pathway (also known as the MAPK pathway) regulates a variety of cellular responses including cell proliferation and differentiation. The pathway uses a cascading effect where RAS (including KRAS variant) activates RAF (including BRAF variant), which in turn activates MEK, which then activates ERK leading to cell growth. However, overexpression or activation of any of the components in the pathway due to activating mutations can lead to tumor growth, as observed with LGSOC.

Primary treatment for LGSOC consists of surgery with comprehensive staging. For patients with stages IC and II-IV disease, adjuvant paclitaxel/carboplatin chemotherapy (± bevacizumab) and/or hormone therapy is recommended, depending on disease stage. For recurrent disease, hormonal therapy or additional chemotherapy are preferred if not previously used. Targeted therapy with an oral MEK inhibitor therapy (e.g., trametinib [Mekinist], binimetinib [Mektovi]) is may also be used for recurrent disease.

Notably, when blocking a single oncogene in the RAS/RAF/MEK/ERK pathway during cancer pharmacotherapy, the tumor may react to treatment by either reactivating the RAS pathway elsewhere or activating a parallel, compensatory pathway to survive. Therefore, drug resistance is associated with agents that target a single point in the RAS/RAF/MEK/ERK pathway.

Unlike existing MEK inhibitors used to treat LGSOC, avutometinib blocks both RAF and MEK, suggesting it may help overcome resistance and inhibit tumor growth and proliferation. In addition, defactinib reduces compensatory signaling of FAK to further combat tumor resistance and tumor growth. In clinical trials, the combination demonstrated a significant response rate in patients with KRAS mutant LGSOC.

There are limited treatment options in LGSOC. If approved, avutometinib plus defactinib will be the first treatment specifically indicated for LGSOC. Verastem is also exploring the use of avutometinib plus defactinib in patients with KRAS wild-type LGSOC, pancreatic cancer and thyroid cancer. The company is also investigating use of avutometinib and/or defactinib in combination with other agents.

FDA approval timeline

June 30, 2025

FDA Designations: Breakthrough Therapy, Orphan Drug, Priority Review, seeking Accelerated Approval

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales for avutometinib only
2025	$13
2026	$25
2027	$48
2028	$74
2029	$108

The financial forecast for defactinib is not currently available.

clesrovimab IM

Manufacturer: Merck

Proposed indications

Respiratory syncytial virus (RSV) disease prevention in infants during their first RSV season

Clinical overview

Mechanism of action

Clesrovimab is a monoclonal antibody with an extended half-life that provides passive immunization for the prevention of RSV disease.

Clinical trial(s)

Clesrovimab was evaluated in the double-blind, Phase 2b/3 CLEVER trial (NCT04767373; n=3,632) in healthy preterm and full-term infants (birth to 1 year of age) during their first RSV season. Patients were randomized 2:1 to clesrovimab or placebo. Most patients in each group (~83%) were late- and full-term infants (gestational age ≥ 35 weeks). Clesrovimab significantly reduced the primary endpoint of incidence of RSV-associated medically attended lower respiratory infections (MALRI) requiring ≥ 1 indicator of lower respiratory infection (LRI) or severity compared to placebo through day 150 (five months) post dose by 60.4% (p<0.001). Clesrovimab also reduced RSV-associated hospitalizations (secondary endpoint) by 84.2% (p<0.001) compared to placebo. Results were consistent at the six-month time point. The safety profile for clesrovimab was like placebo.

Clesrovimab is also being evaluated in the ongoing, partially blinded, controlled, Phase 3 SMART trial (NCT04938830) in infants and children at increased risk for severe RSV disease due to prematurity (≤ 35 weeks gestational age), chronic lung disease (CLD) of prematurity, or hemodynamically significant congenital heart disease (CHD). Patients were randomized 1:1 to clesrovimab or to monthly doses of palivizumab. Interim analysis (n=901) based on data from the patients’ first RSV season revealed a comparable safety profile (primary endpoint) between clesrovimab and palivizumab. The incidence rates of the secondary endpoints of RSV-associated MALRI requiring ≥ 1 indicator of LRI or severity and RSV-associated hospitalizations through day 150 (five months) were also comparable between clesrovimab (3.6% and 1.3%, respectively) and palivizumab (3% and 1.5%, respectively). The SMART trial continues into the second RSV season for eligible participants. Second-season data are not available yet.

Dosage and administration

In the clinical trials, clesrovimab was administered as a single 105 mg IM injection during the first RSV season. In the SMART trial, for eligible patients who received clesrovimab during their second RSV season, the clesrovimab dose is 210 mg IM.

Place in therapy

RSV is a common contagious respiratory virus that usually causes mild, cold-like symptoms lasting 1 to 2 weeks. In the U.S. each year, RSV infection leads to approximately 2.1 million outpatient visits and between 58,000 to 80,000 hospitalizations in children < 5 years of age. Children who are at particular risk for serious RSV infections are premature infants, children < 2 years of age with CLD or CHD of prematurity, and children who are immunocompromised or have neuromuscular disorders.

The RSV season in the U.S. typically occurs during November through April but may vary by region. Treatment for mild cases consists of symptom management, including use of antipyretics and analgesics. Ribavirin inhalation solution (Virazole) is approved in the U.S. to treat hospitalized infants and young children with severe lower respiratory tract disease (LRTD) due to RSV. For prevention of RSV-related LRTD, the FDA has approved the RSV vaccine Abrysvo that is administered during pregnancy in provide protection against RSV-related LRTD in infants from birth through 6 months of age. Abrysvo is also FDA approved for use in adults at increased risk for RSV-related LRTD. In addition, two RSV monoclonal antibodies, nirsevimab-alip (Beyfortus) and palivizumab (Synagis), are approved in infants and young children (up to 24 months of age) who are at risk for serious RSV LRTD. Of the two antibody agents, Beyfortus is preferred by the CDC and American Academy of Pediatrics based on its efficacy, duration, and convenience. Beyfortus is indicated in neonates and infants born during or entering their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. It is administered as a single IM weight-based dose during the first RSV season and as two injections in the second RSV season. Synagis is indicated for use during the first RSV season in patients with premature birth (≤ 35 weeks gestational age) and during the first and second RSV seasons in select children with bronchopulmonary dysplasia (BPD) or CHD. Synagis is administered as a weight-based dose for up to five consecutive monthly IM doses. Neither Beyfortus nor Synagis are indicated for the treatment of RSV infection.

If approved, clesrovimab will be the third monoclonal antibody indicated to prevent RSV infection in pediatric patients. It could compete directly with Beyfortus as the preferred agent in infants at increased risk for RSV infection during their first RSV season. Unlike Beyfortus, the dosage for clesrovimab is the same for all patients during the first RSV season, regardless of their weight. Moreover, the FDA submission for clesrovimab includes data in healthy full-term infants and could expand use of anti-RSV monoclonal antibodies beyond just high-risk infants to include all infants. Merck anticipates clesrovimab to be available for the 2025–26 RSV season if FDA approval is granted.

FDA approval timeline

June 10, 2025

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales
2025	$7
2026	$20
2027	$35
2028	$49
2029	$59

condoliase intervertebral disc injection

Manufacturer: Ferring / Seikagaku

Proposed indications

Radicular leg pain associated with lumbar disc herniation (LDH)

Clinical overview

Mechanism of action

Condoliase, also known as chondroitin sulfate ABC endolyase, is an enzyme that degrades the central (nucleus pulposus) tissue of the vertebral discs to relieve pressure on spinal nerves affected by disc herniation.

Clinical trial(s)

Condoliase was evaluated in two double-blind, sham/placebo-controlled, Phase 3 trials in adults with radicular leg pain associated with LDH. The first trial (NCT03607838) was conducted in the U.S. and included 341 patients in the modified ITT populations. The second trial was conducted in Japan and included 163 patients with LDH. Both trials enrolled patients who failed to achieve resolution of pain with at least six weeks of conservative therapy. In both studies, condoliase compared to sham/placebo met the primary endpoint of change from baseline to week 13 in worst leg pain during the past 24 hours averaged over the previous seven days as measured by the Visual Analog Scale (pain rating score range, 0 to 100) (LS mean difference, -7.5 [p=0.0263] in the U.S. trial and -15.2 [p=0.001] in the Japan trial). However, in the U.S. study, condoliase did not result in a significant change in average worst leg pain at week 52. Data at 52 weeks were not reported for the Japan study.

Dosage and administration

In the clinical trials, condoliase was administered as a single 1.25 unit intradiscal injection

Place in therapy

Herniation of spinal lumbar discs is the most common cause of low back and leg pain. Disc herniation occurs when the jelly-like center (nucleus pulposus) of the disc presses against its outer ring, putting pressure on spinal nerves and causing pain in the lower back, buttocks, and legs. Herniation may be caused by age-related degeneration or injury to the disc. Risk factors include improper lifting, overweight or obesity, repetitive movements of the spine, prolonged sitting or standing, and smoking. The annual incidence of a herniated disc is estimated as 5 to 20 cases per 1,000 adults. It occurs most often during the third to fifth decade of life and men are twice as likely as women to experience a herniated disc.

Symptoms associated with an acute herniated disc will resolve within 8 to 12 weeks without any specific treatment in the majority (85%) of affected patients. Conservative, non-surgical treatments, including NSAIDs and physical therapy (after initial onset of symptoms) to strengthen lower back and abdominal muscles, are usually first-line treatment. For cases that do not respond to OTC medications, short-term use of opioids may be considered; however, routine use of opioid agents for chronic low back pain is not recommended. Second-line treatment includes epidural corticosteroid injections and nerve root blocks. Use of duloxetine, tricyclic antidepressants, and tramadol has also been commonly reported for pain. Lastly, surgical procedures for an LDH, including laminectomies and discectomies, may be required to relieve pressure on the spinal nerves.

In clinical trials, condoliase intradiscal injection led to significant improvement in worst leg pain after 13 weeks. If approved, it will provide a less invasive option for treatment of radicular leg pain associated with LDH compared to surgical procedures in patients who failed conservative therapy. Notably, condoliase is isolated and purified from Gram-negative Proteus vulgaris bacteria. The body, therefore, considers condoliase a foreign protein, and use is limited to a once in a lifetime injection to prevent anaphylactic reactions.

On January 10, 2025, the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee voted 8 to 4 in favor that condoliase’s benefits outweighed its risks in patients with radicular leg pain associated with LDH. The panel acknowledged that condoliase would provide a less invasive option compared to surgery, if approved. However, panel members had concerns related safety profile, such as increased risks for severe cutaneous adverse reaction [SCAR], arthritis and disc degeneration. Additional concerns were related to lack of long-term data and the need for special training for clinicians. Panel members recommended a narrower indication, including confirmation of nerve compression, prior failure of six weeks of conservative therapy, and post-marketing surveillance.

FDA approval timeline

March 2025

Financial forecast (reported in millions)

The financial forecast for condoliase is not currently available.

etripamil (Cardamyst) intranasal

Manufacturer: Milestone

Proposed indications

Paroxysmal supraventricular tachycardia (PSVT)

Clinical overview

Mechanism of action

Etripamil is a fast-acting, intranasal non-dihydropyridine calcium channel blocker (CCB) for the acute conversion of atrioventricular (AV)-nodal-dependent PSVT.

Clinical trial(s)

The double-blind, placebo-controlled, Phase 3 RAPID trial (NCT03464019) evaluated etripamil in adults with a history of PSVT with sustained, symptomatic episodes (≥ 20 minutes) as documented by electrocardiogram (ECG). Patients who tolerated test doses of etripamil during sinus rhythm were randomized to receive either etripamil or placebo. In the study, 99 patients assigned to etripamil, and 85 patients assigned to placebo experienced a confirmed AV-nodal-dependent episode of PSVT. The primary endpoint was time to conversion of PSVT to sinus rhythm for at least 30 seconds within 30 minutes after the first study dose, as recorded by ECG. The study reported that significantly more patients in the etripamil group met the primary endpoint compared to the placebo group (64% versus 31%, respectively; p<0.0001). The median time to conversion was 17.2 minutes with etripamil compared to 53.5 minutes with placebo. In addition, 66% percent of patients in the etripamil group administered a second study dose due to persistent symptoms compared to 79% in the placebo group. The recurrence rate of PSVT after conversion (up to 5 hours post dose) was low in each group (3% with etripamil, 4% with placebo). Primary efficacy outcomes did not differ based on age or concomitant use of cardiac medications (e.g., CCB, beta blocker). While a lower percentage of patients in the etripamil group sought additional medical interventions and emergency department visits compared to the placebo group, the difference was not significant. No serious adverse events were reported. The most common TEAEs were nasal discomfort, nasal congestion and rhinorrhea.

Dosage and administration

In the RAPID clinical trial, patients self-administered on-demand doses of etripamil 70 mg nasal spray. Patients were trained to recognize onset of PSVT symptoms, at which time the patient would attach an ECG monitor and conduct the vagal maneuver. If symptoms did not resolve, the patient administered their assigned study treatment (etripamil or placebo). Patients administered a second dose if PSVT symptoms persisted at 10 minutes. If symptoms did not resolve within 30 minutes of the first dose, patients sought appropriate care.

Place in therapy

PSVT is characterized by a fast (> 100 beats per minute [bpm]) and irregular heartbeat with abrupt onset and termination. It affects the upper chambers of the heart (atria) and prevents the heart from adequately pumping blood. Symptoms include dizziness, palpitations, chest discomfort, shortness of breath, sweating and fainting. Frequent and untreated episodes may eventually cause heart failure and severe episodes that may lead to sudden cardiac arrest. Risk factors for PSVT include heart disease, obstructive sleep apnea, thyroid disease, uncontrolled diabetes, stress, middle age or older, excessive caffeine or alcohol intake, and smoking. It is estimated that about 570,000 persons in the U.S. are living with PSVT with about 89,000 new cases occurring each year. Women are twice as likely as men to develop PSVT.

Overall, supraventricular tachycardia, including atrial and ventricular tachycardia, accounts for approximately 50,000 emergency department visits each year. A joint guidance by the American College of Cardiology, American Heart Association, and Heart Rhythm Society (2015) recommends vagal maneuvers (e.g., coughing, holding the nostrils closed while attempting to blow air through the nose, cold compress on face), carotid sinus massage and/or IV adenosine for acute treatment of regular PSVT episodes. If this initial therapy is ineffective or not possible, then IV administration of a beta blocker or non-dihydropyridine CCB is recommended in patients who are hemodynamically stable, and synchronized cardioversion is recommended for those who are hemodynamically unstable. For ongoing management of symptomatic PSVT, ablation therapy provides a definitive cure (rate > 95%). In patients who decline or are not candidates for ablation therapy, chronic medical therapy with an oral beta blocker or non-dihydropyridine CCB is recommended. The agencies also advise that self-administered (“pill-in-the-pocket”) off-label use of acute doses of an oral beta-blocker or non-dihydropyridine CCB may be reasonable for ongoing management to terminate acute episodes of PSVT caused by AV-nodal reentrant tachycardia. It is important to note that the beta blocker landiolol (Rapiblyk) was FDA approved in November 2024 and is not included in these guidelines. Rapiblyk is administered IV for the short-term reduction of ventricular rate in adults with supraventricular tachycardia (SVT).

Current guidelines recommend that in select cases, personalized, self-directed interventions, such as vagal maneuvers and “pill-in-the-pocket” drug therapy, can be employed for on-demand treatment of PSVT. Etripamil will be the first agent specifically indicated for self-administered, on-demand treatment of PSVT if approved. In clinical trials, it provided rapid resolution of PSVT and was associated with fewer medical interventions and emergency department visits compared to placebo.

FDA approval timeline

March 26, 2025

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales
2025	$6
2026	$27
2027	$87
2028	$128
2029	$169

fitusiran SC

Manufacturer: Sanofi / Alnylam

Proposed indications

Prophylactic treatment of hemophilia A or B in adults and adolescents with or without inhibitors

Clinical overview

Mechanism of action

Fitusiran is a small interference RNA (siRNA) agent designed to lower antithrombin. It uses the body’s natural cellular RNA interference mechanisms to increase thrombin generation and rebalance hemostasis.

Clinical trial(s)

The open-label, Phase 3 ATLAS-A/B trial (NCT03417245) evaluated fitusiran in 120 men ≥ 12 years of age with severe hemophilia A or B without inhibitors. Enrolled patients experienced a minimum of six bleeding episodes requiring factor concentrate treatment within the last six months prior to screening. Patients were randomized 2:1 to receive fitusiran prophylaxis or to continue on-demand clotting factor concentrates for a total of nine months. The estimated mean annualized bleeding rate (ABR) (primary endpoint) was significantly lower with fitusiran compared to on-demand treatment (3.1 versus 31, respectively; p<0.0001). In addition, 51% of patients in the fitusiran group had zero treated bleeds compared with 5% in the on-demand group. Patients who received fitusiran also reported significant improvement in QOL. The most common TEAE reported with fitusiran was ALT elevation (23%). A serious TEAE was reported in 6% of patients who received fitusiran, including cholelithiasis, cholecystitis, lower respiratory tract infection and asthma.

Fitusiran was evaluated in the open-label, Phase 3 ATLAS-INH trial (NCT03417102) in 57 men ≥ 12 years of age with severe hemophilia A or B with inhibitors. Enrolled patients experienced at least six bleeding episodes requiring treatment with a bypassing agent (BPA) within the last six months prior to screening. Patients were randomized 2:1 to receive either fitusiran prophylaxis or on-demand treatment with a BPA for a total of nine months. The study reported a 90.8% reduction in the primary endpoint of ABR with fitusiran prophylaxis compared to an on-demand BPA (ABR, 1.7 versus 18.1, respectively; p<0·0001). In addition, 25 (66%) patients in the fitusiran group experienced zero bleeds requiring treatment compared to one (5%) patient in the BPA group. Significant improvement in QOL measures were also reported with fitusiran. The most common TEAE reported with fitusiran was ALT elevation (32%). Suspected or confirmed thromboembolic events were reported in two (5%) patients who received fitusiran.

Interim data from the open-label, Phase 3 ATLAS-PPX trial were consistent with ATLAS-INH and ATLAS-A/B trials. ATLAS-PPX enrolled male patients ≥ 12 years of age with severe hemophilia A or B with or without inhibitors who were previously treated with factor or BPA prophylaxis. In the study, all patients switched from their prior therapy to fitusiran prophylaxis for seven months. The study reported a significant 61.1% (p=0.0008) reduction in ABR with fitusiran prophylaxis compared with factor or BPA prophylaxis. In addition, 63.1% of patients treated with fitusiran experienced zero treated bleeds compared to 16.9% of patients with prior factor or BPA prophylaxis.

Patients with hemophilia are at increased risk during surgery; therefore, it is necessary patients undergoing major surgery to have options to maintain hemostasis. Major surgeries conducted during the fitusiran clinical development program were evaluated. Analysis included 60 major surgeries, including 24 among patients with factor inhibitors. Data revealed major surgeries were safely and effectively performed following bleed management guidelines in patients with hemophilia treated with fitusiran, regardless of inhibitor status.

Dosage and administration

In the clinical trials, fitusiran 80 mg was administered as a SC injection once a month. Bypass agents were administered as needed for breakthrough bleeding episodes up to a total of nine months.

Place in therapy

Hemophilia is an X-linked congenital bleeding disorder that affects up to 33,000 men in the U.S; it occurs rarely in women. The condition is characterized by chronic spontaneous bleeding into muscles and joints that can progress to debilitating arthropathy. Hemophilia A and B exhibit low or missing levels of clotting factors VIII and IX, respectively. Hemophilia A comprises about 80% of all cases.

The main treatment for the management of severe hemophilia is factor replacement therapy with plasma-derived or recombinant clotting factor concentrates (CFCs). However, approximately 35% of patients with hemophilia A and 3% with hemophilia B will develop neutralizing antibodies, or inhibitors, to factor products, making the condition more difficult to treat. BPAs contain certain clotting factors that circumvent the factor inhibitor to allow the body to form a normal clot and stop bleeding. BPAs, including recombinant activated factor VIIa (e.g., Novoseven RT, Sevenfact) and activated prothrombin complex concentrates (e.g., Feiba), are the SOC to reduce bleeding and long-term complications of hemophilia in patients with inhibitors. Hemostatic efficacy rates of about 80% have been reported with BPAs but may vary and change over time.

Non-factor products have been developed which improve hemophilia management, including in patients with factor inhibitors, and may also minimize treatment burden and improving QOL. Available non-factor products include the bispecific FIXa/FX antibody emicizumab-kxwh (Hemlibra), which mimics FVIIIa cofactor. Hemlibra is indicated for routine prophylaxis for patients of any age with hemophilia A with or without inhibitors and is self-administered SC every 1, 2, or 4 weeks. The anti-tissue factor pathway inhibitors, concizumab-mtci (Alhemo) and marstacimab-hncq (Hympavzi), are approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ≥ 12 years of age with hemophilia A or B; the agents are approved for patients with and without inhibitors, respectively. While Alhemo maintenance therapy is self-administered as once-daily SC injections, Hympavzi maintenance doses are self-administered SC once weekly. In addition, the FDA approved gene therapies for hemophilia, including valoctocogene roxaparvovec-rvox (Roctavian) for select adults with severe hemophilia A without factor VIII inhibitors and fidanacogene elaparvovec-dzkt (Beqvez) for certain adults with moderate to severe hemophilia B. Both gene therapies are administered as a one-time IV infusion.

Fitusiran is a first-in-class siRNA agent for hemophilia. If approved, it will provide an alternative non-factor prophylactic treatment for patients with hemophilia A or B with or without inhibitors. In Phase 3 trials, once monthly SC injections significantly reduced ABR, with ABR rates of zero observed for more than half of patients. In addition, interim results from the open-label extension ATLAS-OLE trial reported benefit with every other month dosing, further reducing treatment burden compared to factor and other non-factor products (excluding gene therapy). In addition, clinical trials showed that major surgeries can be safely and effectively conducted in patients taking fitusiran when following bleed management guidelines.

FDA approval timeline

March 28, 2025

FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales
2025	$33
2026	$75
2027	$110
2028	$131
2029	$149

pegzilarginase IV, SC

Manufacturer: Immedica

Proposed indications

Arginase 1 deficiency (ARG1-D)

Clinical overview

Mechanism of action

Pegzilarginase is a recombinant, cobalt-substituted and pegylated human arginase 1 (ARG1) enzyme that lowers levels of arginine and its toxic metabolites in the plasma. The cobalt-substitution allows for increased catalytic activity compared to the natural arginase enzyme.

Clinical trial(s)

The double-blind, placebo-controlled, Phase 3 PEACE trial (NCT03921541) evaluated efficacy and safety of pegzilarginase added to SOC (protein-restricted diet) in patients ≥ 2 years of age with ARG1-D. A total of 32 patients were randomized 2:1 to pegzilarginase or placebo. After 24 weeks, pegzilarginase led to a significantly greater reduction in geometric mean plasma arginine (pArg) levels compared to placebo (76%; p<0.0001); notably, there were no meaningful changes in arginine levels reported with placebo. At 24 weeks, 90.5% of patients treated with pegzilarginase achieved a pArg level < 200 μmol/L (normal range, 100 to 200 μmol/L). In the pegzilarginase arm, the mean pArg was within normal range (40 to 115 μmol/L) by week 12. Key secondary outcomes of Gross Motor Function Measure part E (GMFM-E) and 2-minute walk test (2MWT) showed numeric but not statistical improvements in functional mobility with pegzilarginase compared to placebo at week 24 (LSM differences, +4.6 points and +5.5 meters, respectively). In addition, patients who continued pegzilarginase during the open-label, long-term extension phase maintained pArg levels within normal range and continued to experience meaningful improvements in mean GMFM-E and 2MWT through week 24 (48 weeks of total treatment). TEAEs were generally mild and transient. The most common TEAEs (≥ 15%) were vomiting, pyrexia, cough and elevated ammonia levels during the double-blind period.

Dosage and administration

In the PEACE trial, pegzilarginase was administered via a 30-minute IV infusion. Treatment was initiated with a dose of 0.1 mg/kg per week with dose adjustments as needed to achieve a pArg between 50 and 150 μmol/L at the end of the dosing interval (168 hours post-dose). The possible dosages ranged from 0.05 to 0.2 mg/kg per week. Patients could switch to SC administration administered by an HCP, using the same formulation and dose level, after the first eight weeks of the long-term extension phase. After the fourth SC dose, patients had the option for doses to be HCP-administered at home.

Place in therapy

ARG1-D is a rare, genetic, metabolic urea cycle disorder characterized by a lack of functional arginase enzyme in the liver and red blood cells. This leads to high levels of arginine in the blood and cerebrospinal fluid. Excess accumulation of ammonia in the blood can also occur. The majority of patients present in early childhood; however, those with a partial enzyme deficiency may become symptomatic in later childhood or in adulthood. Frequent vomiting and poor appetite with food refusal and protein aversion are common. If left untreated, ARG1-D leads to spasticity, gait disorders, difficulty walking, developmental delay, failure to thrive, and seizures. ARG1-D is estimated to occur in approximately 1 in 300,000 to 1,000,000 births and can be identified at birth through newborn screening. 

Management of ARG1-D consists of a life-long severe protein restricted diet to reduce pArg levels and essential amino acids supplementation. Guidelines recommend lowering pArg to less than 200 μmol/L but this is rarely achieved with dietary restriction due to endogenous arginine sources. Ammonia scavenger medications (e.g., sodium phenylacetate, sodium benzoate) to clear accumulated ammonia from the blood may also be used. Arginine and ammonia levels should be monitored periodically, and prompt treatment (e.g., IV fluids and ammonia scavengers, mannitol [for cerebral edema]) should be administered for excess levels. Even with current management approaches, most patients develop moderate to severe neurological damage, leading to poor quality of life and decreased lifespan. 

If approved, pegzilarginase will be the first medication that reduces pArg to normal levels, fulfilling a large unmet need in treating ARG1-D. However, the PEACE trial did not demonstrate a statistically significant improvement in functional mobility at 24 weeks with pegzilarginase treatment. Failure to meet statistical significance may have been influenced by differences among patients’ disease severity, disease duration, and functional performance at baseline that may have led to variability in functional response. Notably, data from the trial suggest that longer-term use of pegzilarginase may lead to clinical stabilization of patients with ARG1-D.

FDA approval timeline

May to July 2025

FDA designations: Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD

Financial forecast (reported in millions)

The financial forecast for pegzilarginase is not currently available.

sebetralstat oral

Manufacturer: Kalvista

Proposed indications

Hereditary angioedema (HAE) on-demand treatment for patients ≥ 12 years of age

Clinical overview

Mechanism of action

Sebetralstat is an oral plasma kallikrein inhibitor.

Clinical trial(s)

The double-blind, placebo-controlled, Phase 3, three-way crossover KONFIDENT trial (NCT05259917) evaluated sebetralstat to treat acute HAE attacks in 136 patients ≥ 12 years of age. Enrolled patients had at least two documented HAE attacks within three months prior to randomization. Those receiving long-term prophylactic treatment for HAE must have been on stable doses for at least three months prior to screening and for the remainder of the trial. The study reported that the primary endpoint of time to the beginning of symptom relief, as recorded using the Patient Global Impression of Change (PGI-C), was significantly faster with sebetralstat 300 mg and sebetralstat 600 mg compared to placebo (median, 1.61 hours, 1.79 hours, and 6.72 hours, respectively; p≤0.001 for both doses). Time to decrease in attack severity was also faster with sebetralstat 300 mg and 600 mg compared to placebo (median, 9.27 hours, 7.75 hours, and > 12 hours, respectively; p=0.004 and p=0.003, respectively). In addition, the percentage of attacks with complete resolution within 24 hours was 42.5% with sebetralstat 300 mg, 49.5% with sebetralstat 600 mg, and 27.4% with placebo. Notably, 39.1% and 39.8% of attacks that were treated with sebetralstat 300 mg or 600 mg, respectively, required a second dose compared to 56% of attacks that were treated with placebo. Additional analysis revealed a correlation between time to treatment and duration of HAE attack. The overall median time to receive treatment was 41 minutes. Complete attack resolution was reached faster (hazard ratio, 2.72) in attacks that were treated "earlier" (within six minutes of onset) with sebetralstat compared with those that were treated "later" (140 minutes or later; a time similar to parenteral on-demand treatments). Notably, the largest proportion of sebetralstat-treated attacks treated “earlier” were mild at baseline, while the largest proportion of attacks treated “later” were considered moderate at baseline. Safety profiles were similar between sebetralstat and placebo.

Dosage and administration

In the clinical trials, sebetralstat was administered orally as 300 mg or 600 mg. Patients were instructed to treat their attacks as early as possible. Patients could administer a second dose at least 3 hours after the first dose for up to two doses to treat a single HAE attack.

Place in therapy

HAE is a rare, dominant autosomal genetic disorder that affects an estimated 1 in 50,000 globally. In the U.S., HAE attacks result in 15,000 to 30,000 emergency department visits each year. Patients with HAE have low levels of endogenous or functional C1 esterase inhibitor (C1-INH). HAE is characterized by recurrent episodes of nonpruritic, nonpitting, SC or submucosal edema involving the skin or mucosal tissues of the upper respiratory and GI tracts. Although swelling can resolve spontaneously in several days, in some cases laryngeal edema can be fatal if left untreated and the pain of GI attacks can be incapacitating. Symptoms may begin as early as 2 years of age and persist throughout life with unpredictable severity and frequency of attacks. It is thought that minor trauma and stress can lead to an attack; however, many attacks occur without any apparent trigger.

C1-INH deficiency or dysfunction leads to uncontrolled increases in plasma kallikrein activity. This, in turn, leads to increased levels of bradykinin, a potent vasodilator that increases vascular permeability resulting in swelling and pain associated with HAE. Management of HAE involves on-demand treatment of acute attacks and prophylaxis to prevent HAE episodes.

Agents for on-demand treatment of HAE attacks target different steps in the HAE pathophysiology. They include the IV-administered C1 esterase inhibitors (Berinert, Ruconest), the SC-administered bradykinin B2 receptor antagonist icatibant (Firazyr), and the SC-administered plasma kallikrein inhibitor ecallantide (Kalbitor). Firazyr, Berinert and Ruconest may be self-administered with proper training. Kalbitor must be administered by an HCP.

If approved, sebetralstat will be the second plasma kallikrein inhibitor indicated to treat acute attacks of HAE. Moreover, it will be the first oral agent for on-demand use for HAE, providing an important option that will minimize treatment burden and may prevent treatment delay.

FDA approval timeline

June 17, 2025

FDA designations: Fast Track, Orphan Drug

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales
2025	$85
2026	$152
2027	$236
2028	$311
2029	$352

sepiapterin oral

Manufacturer: PTC Therapeutics

Proposed indications

Phenylketonuria (PKU)

Clinical overview

Mechanism of action

Sepiapterin reduces phenylalanine levels in the blood via a dual mechanism of action. Sepiapterin undergoes intracellular conversion to tetrahydrobiopterin (BH4), a critical cofactor of the phenylalanine hydroxylase enzyme. In addition, sepiapterin acts as a pharmacological chaperone that corrects phenylalanine hydroxylase misfolding, resulting in improved enzyme function.

Clinical trial(s)

Sepiapterin was evaluated in the ongoing, two-part, double-blind, placebo-controlled, Phase 3 APHENITY trial in patients with PKU with a blood phenylalanine concentration of ≥ 360 μmol/L. In part one, all patients (n=157) received open-label treatment with sepiapterin for 14 days. Patients ≥ 2 years of age who responded to sepiapterin (n=114), defined as ≥ 15% reduction in blood phenylalanine levels, proceeded to Part 2 of the study. In Part 2, patients were randomized 1:1 to continue treatment with sepiapterin or switch to placebo for six weeks. In the overall primary analysis population, the study reported a significant reduction in the primary endpoint of mean change from baseline in blood phenylalanine after six weeks with sepiapterin compared to placebo (63% versus 1%, respectively; p<0.0001). The mean change from baseline in blood phenylalanine after six weeks with sepiapterin compared to placebo was also significant among a subgroup of patients with classical PKU (69% versus 3%, respectively; p<0.001). The mean absolute reductions in blood phenylalanine levels in the overall study population with sepiapterin and placebo were 410 and 16 μmol/L, respectively (p<0.0001). Likewise, in patients with classical PKU, the reductions in phenylalanine and placebo were 523 and 42 μmol/L, respectively (p<0.001). Notably, 84% of patients in the study achieved U.S. guideline target blood levels (< 360 μmol/L). Sepiapterin was well tolerated. The most common TEAEs reported with sepiapterin were headache and diarrhea.

An open-label, three-period crossover, active-controlled, Phase 2 trial conducted outside the U.S. compared sepiapterin and saptropterin dihydrochloride (Kuvan) in 24 adults with PKU and hyperphenylalanemia. Patients received seven days of once-daily oral sepiapterin 20 mg/kg/day, sepiapterin 60 mg/kg/day and Kuvan 20 mg/kg/day. Treatment regimens were separated by a 7-day washout. The study reported a LSM change from baseline in blood phenylalanine of -146.9 μmol/L with sepiapterin 20 mg/kg (p=0.001), -206.4 μmol/L with sepiapterin 60 mg/kg (p<0.0001), and -91.5 μmol/L with Kuvan 20 mg/kg/day (p=0.0339) in the overall study population. Sepiapterin 60 mg/kg resulted in a significantly greater decrease in phenylalanine compared to Kuvan 20 mg/kg/day (p=0.0098); the same was not true when comparing sepiapterin 20 mg/kg to Kuvan 20 mg/kg/day. In addition, blood phenylalanine levels < 360 μmol/L were achieved in 46% of patients on sepiapterin 20 mg/kg, 50% on sepiapterin 60 mg/kg, and 42% on Kuvan 20 mg/kg/day. Notably, among patients with classical PKU, LSM changes in blood phenylalanine were -71.5 μmol/L with sepiapterin 20 mg/kg, -150.8 μmol/L with sepiapterin 60 mg/kg, and -2.8 μmol/L with Kuvan 20 mg/kg/day.

Dosage and administration

In the APHENITY trial, sepiapterin was administered orally as a forced-dose escalation of 20, 40, and 60 mg/kg per day per consecutive two-week period. Sepiapterin was provided as a powder for oral use that was suspended in water or apple juice prior to administration.

Place in therapy

PKU is an autosomal recessive inherited metabolic disorder. The overall incidence in the U.S. is approximately one in 10,000 to 15,000 individuals and is higher among Caucasians and Native Americans compared to African American, Hispanic and Asian populations. PKU is caused by mutations in the gene encoding the phenylalanine hydroxylase enzyme. This leads to an inability of the body to metabolize the essential amino acid phenylalanine found in dietary protein. Phenylalanine accumulates to toxic levels that are damaging to the brain. If left untreated, microcephaly, delayed developmental milestones, behavior problems, and seizures, among other complications, can develop. Severity of the illness varies from mild to severe depending on specific gene mutations causing the disease and the level of functional phenylalanine hydroxylase present. Classical PKU is the most severe form of the disease and is characterized by blood phenylalanine concentrations > 1,200 μmol/L with normal dietary protein intake.

In the U.S., PKU is typically detected at birth during newborn screening. Life-long adherence to a phenylalanine (protein)-restricted diet is necessary to prevent severe cognitive impairments. The amount of tolerated dietary phenylalanine varies between individual patients depending on the PKU severity. Blood phenylalanine levels in all patients should be maintained in the range of 120 to 360 µmol/L. For patients with moderate to severe PKU, medical foods that provide essential amino acids are also necessary for adequate growth and development.

While a phenylalanine-restricted diet may be effective at managing blood phenylalanine levels, continual adherence may be difficult and often wanes during adolescence and early adulthood. In addition, phenylalanine levels may fluctuate during periods of growth, pregnancy, and illness. The FDA has approved two medications to reduce blood alanine levels in patients with PKU to be used in conjunction with a phenylalanine-restricted diet. Pegvaliase-pqpz (Palynziq), a phenylalanine-metabolizing enzyme, is indicated in adults with uncontrolled phenylalanine blood levels (> 600 μmol/L) on existing management. Palynziq maintenance doses are self-administered SC once daily using an autoinjector. The agent carries a boxed warning for the risk of anaphylaxis which may occur at any time during treatment. The phenylalanine hydroxylase activator sapropterin dihydrochloride (Kuvan) is a synthetic form of BH4 and is indicated in patients ≥ 1 month of age with BH4-responsive PKU. Kuvan is available in oral tablets or as a powder for oral solution for once daily administration.

If approved, sepiapterin will provide an additional option for patients of any age with PKU. It is expected to compete with oral Kuvan, which has a limited number (three) of generic manufacturers.

FDA approval timeline

July 2025

FDA designations: Orphan Drug

Financial forecast (reported in millions)

Year	Projected yearly U.S. sales
2025	$14
2026	$76
2027	$134
2028	$196
2029	$250

Keep on your radar

Notable agents that are further from approval have been identified in this unique watch list. These are products with the potential for significant clinical and financial impact. Their development status is being tracked on the Quarterly Drug Pipeline radar. These pipeline products, their respective class or proposed indication, as well as an estimated financial forecast for the year 2029, are displayed. The financials are projected total annual U.S. sales, reported in millions.

Name	Manufacturer	July 2024 pipeline - Total U.S. sales for 2028 (Dollars in millions)
aficamten	Cardiovascular	$1,384
brensocatib	Respiratory	$1,684
chenodiol	Neurometabolic	$94
deramiocel	Cardiovascular / cellular therapy	$122
donidalorsen	Hematology	$287
nipocalimab	Immunology	$705
paltusotine	Endocrine	$424
plozasiran	Cardiovascular	$286
prademagene zamikeracel (EB-101)	Dermatology / gene therapy	$234
rebisufligene etisparvovec (UX111)	Metabolic / gene therapy	$15
rilzabrutinib	Immunology	$257
taletrectinib	Oncology	$194
telisotuzumab vedotin	Oncology	$293
vatiquinone	Neurology	$35
vusolimogene oderparepvec (RP1)	Oncology / gene therapy	$319
zopapogene imadenovec (PRGN-2012)	Oncology / gene therapy	$335

Drug list

The pipeline drug list is an aerial outline of drugs with anticipated FDA approval through 2026. It is not intended to be a comprehensive inventory of all drugs in the pipeline; emphasis is placed on drugs in high-impact categories. Investigational drugs with a recent Complete Response Letter (CRL) are also reported.

Gene and cellular therapies

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
revakinagene taroretcel (NT-501)	Neurotech	Macular telangiectasia type 2	Intravitreal implant	BLA; Fast Track, Orphan Drug, Priority Review	03/18/2025
prademagene zamikeracel (EB-101)	Abeona	Epidermolysis bullosa	Surgical application	BLA; Breakthrough Therapy, Orphan Drug, RMAT, RPD	04/29/2025
rebisufligene etisparvovec (UX111)	Ultragenyx	Mucopolysaccharidosis IIIA (Sanfilippo syndrome type A)	IV	BLA; seeking Accelerated Approval; Fast Track, Orphan Drug, RMAT, RPD	Jul-Dec 2025
zopapogene imadenovec (PRGN-2012)	Precigen	Recurrent respiratory papillomatosis	SC	BLA; seeking Accelerated Approval; Breakthrough Therapy, Orphan Drug	Jul-Dec 2025
vusolimogene oderparepvec (RP1)	Replimune	Melanoma (in combination with nivolumab, prior anti-PD1 regimen)	Intratumoral	BLA; seeking Accelerated Approval; Breakthrough Therapy, Orphan Drug	09/21/2025
deramiocel	Nippon Shinyaku	DMD cardiomyopathy	IV	NDA; Orphan Drug, RMAT, RPD	01/02/2026

None

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
AAV8-ranibizumab (RGX-314)	Abbvie	DME; wet AMD	Subretinal	BLA; Orphan Drug	TBD
aglatimagene besadenovec (CAN-2409)	Candel	Prostate cancer	Intratumorally	BLA; Fast Track	TBD
bidridistrogene xeboparvovec (SRP-9003)	Sarepta	Limb-girdle muscular dystrophy	IV	BLA; may seek Accelerated Approval, Fast Track, Orphan Drug, RPD	TBD
botaretigene sparoparvovec	Janssen	Retinitis pigmentosa	Subretinal	BLA; Fast Track, Orphan Drug	TBD
donaperminogene seltoplasmid (Engensis)	Helixmith	PAD	IM	BLA	TBD
giroctocogene fitelparvovec	Pfizer	Hemophilia A	IV	BLA; Fast Track, Orphan Drug, RMAT	TBD
marnetegragene autotemcel (Kresladi)	Rocket	Leukocyte adhesion deficiency type I (LAD-I)	IV	BLA; Fast Track, Orphan Drug, RMAT, RPD	TBD
OCU400	Ocugen	Retinitis pigmentosa	Subretinal	BLA; Orphan Drug, RMAT	TBD
pariglasgene brecaparvovec (DTX401)	Ultragenyx	Glycogen storage disease (type 1a)	IV	BLA; Fast Track, Orphan Drug	TBD
RGX-121	Regenxbio	Mucopolysaccharidosis II (Hunter syndrome)	Intrathecal	BLA; Fast Track, Orphan Drug, RMAT, RPD	TBD
rilparencel (ReACT)	Prokidney	CKD	Percutaneous (contralateral kidney)	BLA; RMAT	TBD

None

Biosimilars

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
tocilizumab (biosimilar to Genentech's Actemra)	Celltrion	RA; giant cell arteritis; systemic sclerosis-associated interstitial lung disease; JIA (polyarticular, systemic); COVID-19; CRS	IV, SC	BLA	January 2025
ustekinumab (biosimilar to Janssen's Stelara)	Bio-Thera Solutions	PSO; PsA; CD; UC	IV, SC	BLA	May-Jun 2025
ustekinumab (biosimilar to Janssen's Stelara)	Hikma	PSO; PsA; CD; UC	SC	BLA	May-Jun 2025
denosumab (biosimilar to Amgen's Prolia)	Teva	Osteoporosis / osteopenia	SC	BLA	Jul-Aug 2025
denosumab (biosimilar to Amgen's Prolia, Xgeva)	Organon / Henlius	Osteoporosis / osteopenia	SC	BLA	Jul-Aug 2025
aflibercept (biosimilar to Regeneron's Eylea)	Celltrion	DME; diabetic retinopathy; macular edema following RVO; wet AMD	Intravitreal	BLA	Pending
insulin aspart (biosimilar to Novo Nordisk's Novolog)	Amphastar	T1DM; T2DM	SC	BLA	Pending
insulin lispro (biosimilar to Eli Lilly's Humalog)	Gan & Lee	T1DM; T2DM	SC	BLA	Pending
trastuzumab (biosimilar to Genentech's Herceptin)	Tanvex	Breast cancer (HER2+); gastric cancer (HER2+)	IV	BLA	Pending

None

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
bevacizumab (biosimilar to Genetech's Avastin)	Essex	DME; wet AMD	Intravitreal	BLA	TBD

None

Specialty

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
gemcitabine-releasing intravesical system (TAR-200)	Taris / Johnson & Johnson	Bladder cancer (non-muscle-invasive, BCG-unresponsive, high-risk)	Intravesical	NDA; Breakthrough Therapy, Fast Track, RTOR	2025
ataluren (Translarna)	PTC	DMD (nonsense mutation)	Oral	NDA; Fast Track, Orphan Drug, Priority Review, RPD	Jan-Mar 2025
bentracimab	SERB	Ticagrelor (Brilinta) reversal	IV	BLA; Breakthrough Therapy, Priority Review	Jan-Mar 2025
apomorphine	Supernus	Parkinson's disease (continuous treatment of motor fluctuations)	SC infusion	NDA	01/31/2025
vimseltinib	Ono	Tenosynovial giant cell tumor	Oral	NDA; Fast Track, Priority Review	02/17/2025
mirdametinib	Springworks	Neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN)	Oral	NDA; Fast Track, Orphan Drug, Priority Review, RPD	02/28/2025
condoliase	Ferring / Seikagaku	Radicular leg pain associated with lumbar disc herniation	Intervertebral disc injection	BLA	March 2025
camrelizumab	Jiangsu Hengrui	HCC (unresectable, 1st-line, in combination with rivoceranib)	IV	BLA; Orphan Drug	03/20/2025
rivoceranib	Elevar	HCC (unresectable, 1st-line, in combination with camrelizumab)	Oral	NDA; Orphan Drug	03/20/2025
diazoxide choline	Soleno	Prader-Willi syndrome (ages ≥ 4 years with hyperphagia)	Oral	505(b)(2) NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review	03/27/2025
fitusiran	Sanofi /Alnylam	Hemophilia A and B (with or without inhibitors)	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD	03/28/2025
linvoseltamab	Regeneron	Multiple myeloma (R/R, 4th-line)	IV	BLA; Fast Track	Apr-Sep 2025
elamipretide	Stealth	Barth syndrome	SC	NDA; Fast track, Orphan Drug, Priority Review, RPD	04/29/2025
nipocalimab	Janssen	Myasthenia gravis (generalized, anti-AChR, anti-MuSK, anti-LRP4)	IV	BLA; Fast Track, Orphan Drug, Priority Review	04/29/2025
pegzilarginase	Immedica	Arginase 1 deficiency (ARG1-D)	IV, SC	BLA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD	May-Jul 2025
clesrovimab	Merck	RSV prevention (infants, during first RSV season)	IM	NDA	06/10/2025
mitomycin (UGN-102)	Urogen	Bladder cancer (low-grade intermediate-risk non-muscle invasive)	Intravesical	505(b)(2) NDA	06/13/2025
sebetralstat	Kalvista	HAE (on-demand, ages ≥ 12 years)	Oral	NDA; Fast Track, Orphan Drug, Priority Review, RPD	06/17/2025
taletrectinib	Nuvation	NSCLC (advanced ROS1-positive, line-agnostic)	Oral	NDA; Breakthrough Therapy, Orphan Drug, Priority Review	06/23/2025
chenodiol	Mirum	Cerebrotendinous xanthomatosis	Oral	NDA; Orphan Drug	06/28/2025
oxylanthanum carbonate	Unicycive	Hyperphosphatemia (in patients with CKD on dialysis)	Oral	505(b)(2) NDA	06/28/2025
avutometinib	Verastem / Pfizer	Ovarian cancer (recurrent KRAS mutant, low-grade, serous, in combination with defactinib, ≥ 1 prior systemic therapy)	Oral	NDA; seeking Accelerated Approval; Breakthrough Therapy, Orphan Drug, Priority Review	06/30/2025
copper histidinate	Zydus	Menkes disease	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review, RPD	06/30/2025
defactinib	Verastem	Ovarian cancer (recurrent KRAS mutant, low-grade, serous, ≥ 1 prior systemic therapy, in combination with avutometinib)	Oral	NDA; seeking Accelerated Approval; Breakthrough Therapy, Orphan Drug, Priority Review	06/30/2025
brensocatib	Insmed	Bronchiectasis	Oral	NDA; Breakthrough Therapy	Jul-Dec 2025
delgocitinib	LEO	Chronic hand eczema (corticosteroid failure/inappropriate)	Topical	NDA; Fast Track, Priority Review	Jul-Dec 2025
dordaviprone	Chimerix	Brain cancer (recurrent H3 K27M-mutant diffuse glioma)	Oral	NDA; seeking Accelerated Approval; Fast Track, Orphan Drug, RPD	Jul-Dec 2025
sunvozertinib	Dizal (Jiangsu)	NSCLC (locally advanced or metastatic, EGFR exon 20 insertion mutation, prior platinum-based chemotherapy)	Oral	NDA; Breakthrough Therapy, Priority Review	07/07/2025
datopotamab deruxtecan (Datroway)	Daiichi Sankyo	NSCLC (locally advanced or metastatic, EGFR-mutated, ≥ 2nd-line)	IV	BLA; seeking Accelerated Approval; Breakthrough Therapy, Priority Review	07/12/2025
sepiapterin	PTC	Phenylketonuria (PKU)	Oral	NDA; Orphan Drug	07/29/2025
donidalorsen	Ionis	HAE (prophylaxis to prevent attack, ages ≥ 12 years)	SC	BLA; Orphan Drug	04/18/2025
leuprolide mesylate (Camcevi), 3-months	Intas	Prostate cancer (palliative treatment)	SC	505(b)(2) NDA	08/29/2025
rilzabrutinib	Sanofi	ITP	Oral	NDA; Fast Track, Orphan Drug	08/29/2025
lecanemab-irmb (Leqembi) SC	Eisai	Alzheimer's disease (mild, weekly maintenance dosing)	SC	BLA	08/31/2025
sodium pyruvate	EmphyCorp	Idiopathic pulmonary fibrosis (IPF)	Intranasal	NDA	09/12/2025
paltusotine	Crinetics	Acromegaly	Oral	NDA; Orphan Drug	09/25/2025
telisotuzumab vedotin	Abbvie	NSCLC (locally advanced/metastatic, EGFR wild type, nonsquamous, c-Met protein overexpression)	IV	BLA; seeking Accelerated Approval; Breakthrough Therapy, Priority Review	09/26/2025
troriluzole	Biohaven	Spinocerebellar ataxia	Oral	505(b)(2) NDA; Fast Track, Orphan Drug	Oct-Dec 2025
sodium chlorite (NP001)	Neuvivo	ALS	IV	NDA; Orphan Drug	10/07/2025
plozasiran	Arrowhead	Familial chylomicronemia syndrome (FCS)	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug	11/18/2025
lerodalcibep	LIB Therapeutics	LDL-C reduction (with ASCVD or high/very high for ASCVD); HeFH; HoFH	SC	BLA	12/16/2025
vatiquinone	PTC	Friedreich's ataxia	Oral	NDA; Fast Track, Orphan Drug	12/19/2025
relacorilant	Corcept	Cushing's syndrome	Oral	NDA; Orphan Drug	12/30/2025

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
fam-trastuzumab deruxtecan-nxki (Enhertu)	Daiichi Sankyo	Breast cancer (unresectable or metastatic, HER2 low or ultralow, ≥ 1 prior endocrine therapy in metastatic setting)	IV	sBLA; Breakthrough Therapy, Priority Review	02/01/2025
avacincaptad pegol (Izervay)	Astellas	Dry AMD-related geographic atrophy (every other month dosing)	Intravitreal	sNDA; Breakthrough Therapy, Fast Track	02/26/2025
brentuximab vedotin (Adcetris)	Pfizer	Large B-cell lymphoma (R/R, in combination with lenalidomide & rituximab)	IV	sBLA; Orphan Drug	March 2025
furosemide (Furoscix)	scPharmaceuticals	CKD-related edema	SC	sNDA	03/06/2025
vutrisiran (Amvuttra)	Alnylam	Transthyretin amyloid cardiomyopathy (ATTR-CM)	SC	sBLA; Orphan Drug, Priority Review	03/23/2025
durvalumab (Imfinzi)	AstraZeneca	Bladder cancer (muscle invasive)	IV	sBLA; Breakthrough Therapy, Priority Review	Apr-Jun 2025
cabozantinib (Cabometyx)	Exelixis	Neuroendocrine tumors (pancreatic & extra-pancreatic tumors, locally advanced/unresectable or metastatic, well or moderately differentiated, ≥ 2nd-line)	Oral	sNDA; Orphan Drug	04/03/2025
dupilumab (Dupixent)	Sanofi	Chronic spontaneous urticaria (ages ≥ 12 years)	SC	sBLA	04/18/2025
guselkumab (Tremfya)	Janssen/Novartis	CD	IV, SC	sBLA	04/18/2025
ipilimumab (Yervoy)	Bristol Myers Squibb	HCC (unresectable, 1st-line, in combination with nivolumab)	IV	sNDA	04/21/2025
nivolumab (Opdivo)	Bristol Myers Squibb	HCC (unresectable, 1st-line, in combination with nivolumab)	IV	sBLA; Orphan Drug	04/21/2025
ranibizumab port delivery system (Susvimo)	Genentech	DME; Diabetic retinopathy	Intravitreal implant	sBLA	May-Jun 2025
mepolizumab (Nucala)	GlaxoSmithKline	COPD (eosinophilic phenotype, add-on maintenance)	SC	sBLA	05/07/2025
esketamine (Spravato)	Janssen	MDD (treatment-resistant, monotherapy)	Intravitreal implant	sNDA; Breakthrough Therapy, Fast Track	05/22/2025
concizumab-mtci (Alhemo)	Novo Nordisk	Hemophilia A and B (without inhibitors)	SC	sBLA; Breakthrough Therapy	05/31/2025
ruxolitinib (Opzelura)	Incyte	Atopic dermatitis (pediatrics)	Topical	sNDA	Jul-Dec 2025
tafasitamab-cxix (Monjuvi)	Incyte	Follicular lymphoma (R/R); Marginal zone lymphoma (R/R)	IV	sBLA; Orphan Drug	Jul-Dec 2025
upadacitinib (Rinvoq)	Abbvie	Giant cell arteritis	Oral	sNDA; Orphan Drug	07/11/2025
glofitamab-gxbm (Columvi)	Genentech	DLBCL (R/R, in combination with gemcitabine & oxaliplatin, prior therapy, ineligible for autologous SCT)	IV	sBLA	07/20/2025
darolutamide (Nubeqa)	Bayer	Prostate cancer (metastatic, hormone-sensitive, in combination with androgen deprivation therapy [ADT])	Oral	sNDA; Fast Track	07/25/2025
daratumumab / hyaluronidase-fihj (Darzalex Faspro)	Johnson & Johnson	Multiple myeloma (newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone [D-VRd], ASCT deferred or ineligible); Multiple myeloma (high-risk, smoldering)	SC	sBLA	07/30/2025
lonapegsomatropin-tcgd (Skytrofa)	Ascendis	Growth hormone deficiency (adults)	SC	sBLA	07/30/2025
dupilumab (Dupixent)	Sanofi	Bullous pemphigoid	SC	sBLA; Orphan Drug	Aug-Oct 2025
golimumab (Simponi)	Janssen	UC (ages ≥ 2 years)	SC	sBLA; Orphan Drug	10/16/2025

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
amlitelimab	Sanofi	Atopic dermatitis	SC	BLA	TBD
amniotic suspension allograft (ReNu)	Organogenesis	Osteoarthritis (knee)	Intra-articular	BLA; RMAT	TBD
apitegromab	Scholar Rock	Spinal muscular atrophy	IV	BLA; Fast Track, Orphan Drug, RPD	TBD
apraglutide	Ironwood	Short bowel syndrome (parenteral support-dependent)	SC	NDA; Orphan Drug	TBD
asciminib	Compass	Biliary tract cancer	IV	BLA; Fast Track	TBD
astegolimab	Genentech / Amgen	COPD	IV	BLA	TBD
atacicept	Vera	IgA nephropathy (Berger's disease)	SC	BLA; Breakthrough Therapy, Orphan Drug	TBD
azetukalner	Xenon	Focal-onset seizures	Oral	NDA	TBD
bevacizumab-vikg	Outlook	Wet AMD	Intravitreal	BLA	TBD
botaretigene sparoparvovec	Janssen	Retinitis pigmentosatd>	Subretinal	BLA; Fast Track, Orphan Drug	TBD
buntanetap	Annovis	Alzheimer's disease; parkinson's disease	Oral	NDA	TBD
cetuximab sarotalocan	Rakuten	SCCHN	IV	BLA; Fast Track	TBD
cobolimab	GlaxoSmithKline	NSCLC (2nd-line)	IV	BLA	TBD
denecimig	Novo Nordisk	Hemophilia A	SC	BLA; Orphan Drug	TBD
deoxythymidine / deoxycytidine	UCB	Thymidine kinase 2 (TK2) deficiency	Oral	BLA; Breakthrough Therapy, Orphan Drug	TBD
depemokimab	GlaxoSmithKline	Asthma; chronic rhinosinusitis	SC	BLA	TBD
dinutuximab beta (Qarziba)	EUSA	Neuroblastoma	IV	BLA; Orphan Drug	TBD
donaperminogene seltoplasmid	Helixmith	PAD	IM	BLA	TBD
fenebrutinib	Genentech	MS	Oral	NDA	TBD
fianlimab	Regeneron	Melanoma	IV	BLA; Fast Track	TBD
frexalimab	Sanofi	MS	IV, SC	BLA	TBD
garetosmab	Regeneron	Fibrodysplasia ossificans progressiva	IV	BLA; Fast Track, Orphan Drug	TBD
gedatolisib	Celcuity / Pfizer	Breast cancer (HR+/HER2-)	IV	NDA; Breakthrough Therapy, Fast Track	TBD
giredestrant	Roche	Breast cancer (HR+/HER2-)	Oral	NDA	TBD
gold nanocrystal	Clene	ALS	Oral	NDA; Orphan Drug	TBD
govorestat	Applied	Sorbitol dehydrogenase (SORD) deficiency	Oral	NDA; Orphan Drug	TBD
hydromethylthionine mesylate	Taurx	Alzheimer's disease (mild-moderate)	Oral	NDA	TBD
hydroxypropyl beta cyclodextrin	Cyclo	Niemann-Pick disease (type C)	IV	NDA; Fast Track, Orphan Drug, RPD	TBD
ianalumab	Novartis	Autoimmune hepatitis; ITP	SC	BLA	TBD
imsidolimab	Anaptysbio	Generalized pustular psoriasis (GPP)	IV, SC	BLA; Orphan Drug	TBD
inaxaplin	Vertex	Focal segmental glomerulosclerosis	Oral	NDA; may seek Accelerated Approval; Breakthrough Therapy	TBD
itepekimab	Regeneron	COPD	SC	BLA; Fast Track	TBD
ketamine (NRX-100)	Hope	Bipolar disorder (suicidal depression)	IV	NDA; Fast Track	TBD
lanifibranor	Inventiva	MASH	Oral	NDA; Breakthrough Therapy, Fast Track	TBD
latozinemab	Alector / GlaxoSmithKline	Frontotemporal dementia	IV, SC	BLA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
ligelizumab	Novartis	Peanut allergy	SC	BLA	TBD
linerixibat	GSK	Cholangitis pruritus	Oral	NDA; Orphan Drug	TBD
molgramostim (Molbreevi)	Savara	Pulmonary alveolar proteinosis	Inhaled	BLA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
molgramostim (Molbreevi)	Savara	Pulmonary alveolar proteinosis	Inhaled	BLA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
navepegritide	Ascendis	Achondroplasia	SC	NDA; Orphan Drug	TBD
nemvaleukin alfa	Mural	Ovarian cancer (platinum-resistant, in combination with pembrolizumab)	IV	BLA; Fast Track	TBD
nerandomilast	Boehringer Ingelheim	Idiopathic pulmonary fibrosis	Oral	NDA; Breakthrough Therapy, Orphan Drug	TBD
obefazimod	Abivax	UC	Oral	NDA	TBD
pabinafusp alfa	JCR	Mucopolysaccharidosis II (Hunter syndrome)	IV	BLA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
palazestrant	Olema	Breast cancer (HR+/HER2-)	Oral	NDA; Fast Track	TBD
pegadricase	Swedish Orphan Biovitrum	Gout	IV	BLA; Fast Track	TBD
pegargiminase	Polaris	Mesothelioma	IM	BLA; Fast Track, Orphan Drug	TBD
pelabresib	Novartis	Myelofibrosis	Oral	NDA; Fast Track, Orphan Drug	TBD
pelacarsen	Novartis	Dyslipidemia	SC	NDA; Fast Track	TBD
potravitug	Memo	BK polyomavirus (BKV) infection	IV	BLA; Fast Track	TBD
remibrutinib	Novartis	MS; urticaria	Oral	NDA	TBD
resiniferatoxin	Grunenthal	Osteoarthritis (knee)	Intra-articular	NDA; Breakthrough Therapy	TBD
riliprubart	Sanofi	Chronic inflammatory demyelinating polyneuropathy (CIDP)	IV, SC	BLA; Orphan Drug	TBD
rusfertide	Takeda	Polycythemia vera	SC	NDA; Fast Track, Orphan Drug	TBD
savolitinib	AstraZeneca	NSCLC (MET+, 2nd-line)	Oral	NDA; Fast Track	TBD
sefaxersen	Roche	IgA nephropathy (Berger's disease)	SC	NDA	TBD
serplulimab	Henlius	SCLC	IV	BLA; Orphan Drug	TBD
sibeprenlimab	Otsuka	IgA nephropathy (Berger's disease)	IV, SC	BLA; Breakthrough Therapy	TBD
soticlestat	Takeda	Dravet syndrome; Lennox-Gastaut syndrome	Oral	NDA; Orphan Drug	TBD
sozinibercept	Opthea	Wet AMD	Intravitreal	BLA; Fast Track	TBD
suramin	Paxmedica	Human African sleeping sickness	IV	NDA; Orphan Drug	TBD
tamibarotene	Syros	Myelodysplastic syndrome	Oral	NDA; Fast Track, Orphan Drug	TBD
tebipenem pivoxil	GSK	UTI (complicated)	Oral	NDA; Fast Track, QIDP	TBD
tiragolumab	Genentech	Esophageal cancer; NSCLC	IV	BLA; Breakthrough Therapy, Fast Track; Orphan Drug	TBD
tividenofusp alfa	Denali	Mucopolysaccharidosis II (Hunter syndrome)	IV	BLA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD	TBD
tolebrutinib	Sanofi	MS	Oral	NDA; Breakthrough Therapy	TBD
veligrotug	Viridian	Thyroid eye disease	IV	BLA	TBD
venglustat	Sanofi	Fabry disease; gaucher disease	Oral	NDA; Fast Track, Orphan Drug	TBD
cabozantinib	Exelixis	Prostate cancer (metastatic, castration-resistant)	Oral	NDA	TBD
ligelizumab	Novartis	Food allergies	SC	BLA	TBD
REGN-5713-5714-5715	Regeneron	Birch allergy	SC	BLA	TBD
rexlemestrocel-L (Revascor)	Mesoblast	Ischemic HFrEF (adults, end-stage, with left ventricular assist device [LVAD] implantation)	Intramyocardial injection	BLA; may seek Accelerated Approval, Orphan Drug, RMAT	TBD

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
aminolevulinic acid HCl (Ameluz)	Biofrontera	Superficial basal cell carcinoma (sBCC)	Topical	sNDA	TBD
atezolizumab (Tecentriq)	Genentech	Prostate cancer (metastatic, castration-resistant, in combination with cabozantinib)	IV	sBLA	TBD
brolucizumab-dbll (Beovu)	Novartis	Diabetic retinopathy	Intravitreal	sBLA	TBD
cabozantinib (Cabometyx)	Exelixis	Prostate cancer (metastatic, castration-resistant)	Oral	sNDA	TBD
cemiplimab-rwlc (Libtayo)	Regeneron	Melanoma	IV	sBLA; Fast Track	TBD
crovalimab-akkz (Piasky)	Genentech	Hemolytic uremic syndrome	IV, SC	sBLA	TBD
eplontersen (Wainua)	Ionis	Transthyretin amyloid cardiomyopathy (ATTR-CM, wild type or hereditary)	SC	sNDA; Fast Track, Orphan Drug	TBD
iptacopan (Fabhalta)	Novartis	C3 glomerulopathy (C3G)	Oral	sNDA; Breakthrough Therapy, Orphan Drug, RPD	TBD
lenacapavir (Sunlenca)	Gilead	HIV-1 infection prevention	Oral, SC	sNDA	TBD
mitapivat (Pyrukynd)	Agios	SCD	Oral	sNDA; Orphan Drug	TBD
mosunetuzumab-axgb (Lunsumio)	Genentech	DLBCL (2nd-line; in combination with polatuzumab vedotin-piiq)	SC	sBLA	TBD
nogapendekin alfa inbakicept-pmln (Anktiva)	Immunitybio	NSCLC	IV, SC	sBLA	TBD
obinutuzumab (Gazyva)	Genentech	Lupus nephritis; SLE	IV	sBLA; Breakthrough Therapy	TBD
pegcetacoplan (Empaveli)	Apellis	C3 glomerulopathy (C3G)	SC	sNDA; Orphan Drug	TBD
retifanlimab-dlwr (Zynyz)	Incyte	Anal cancer	IV	sBLA; Fast Track, Orphan Drug	TBD
ropeginterferon alfa-2b (Besremi)	PharmaEssentia	Essential thrombocythemia	SC	sBLA; Orphan Drug	TBD
secukinumab (Cosentyx)	Novartis	Giant cell arteritis; lupus nephritis	SC	sBLA	TBD
sparsentan (Filspari)	Travere	Focal segmental glomerulosclerosis	Oral	sNDA; Orphan Drug	TBD
tislelizumab-jsgr (Tevimbra)	Beigene	HCC	IV	sBLA; Orphan Drug	TBD
venetoclax (Venclexta)	Abbvie	Myelodysplastic syndrome	Oral	sNDA; Breakthrough Therapy, Orphan Drug	TBD
zanidatamab-hrii (Ziihera)	Jazz	Gastroesophageal adenocarcinoma (HER2+)	IV	sBLA; Fast Track, Orphan Drug	TBD

Traditional

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
suzetrigine	Vertex	Acute pain (moderate to severe)	Oral	NDA; Breakthrough Therapy, Fast Track, Priority Review	01/30/2025
meloxicam / rizatriptan	Axsome	Migraine (acute treatment)	Oral	505(b)(2) NDA	01/31/2025
chikungunya vaccine (CHIKV VLP)	Bavarian Nordic	Chikungunya virus immunization (ages ≥ 12 years)	IM	BLA; Breakthrough Therapy, Fast Track, Priority Review	02/14/2025
meningococcal vaccine (MenABCWY)	GlaxoSmithKline	Meningococcal immunization	IM	BLA	02/14/2025
hydrocortisone oral solution (ET-400)	Eton	Adrenocortical insufficiency	Oral	NDA	02/28/2025
etripamil (Cardamyst)	Milestone	Paroxysmal supraventricular tachycardia (PSVT)	Intranasal	NDA	03/26/2025
gepotidacin	GlaxoSmithKline	UTI (uncomplicated, females, ages ≥ 12 years)	Oral	NDA; Priority Review, QIDP	03/26/2025
NVX-CoV2373	Novavax	COVID-19 immunization	IM	BLA	April 2025
reproxalap	Aldeyra	DED	Ophthalmic	NDA	04/02/2025
dihydroergotamine	Shin Nippon	Acute migraine	Intranasal	505(b)(2) NDA	04/30/2025
atrasentan	Novartis	IgA nephropathy (Berger's disease)	Oral	NDA; seeking Accelerated Approval	May-Jun 2025
AR-15512	Aerie	DED	Ophthalmic	NDA	05/30/2025
COVID-19 vaccine (mRNA-1283)	Moderna	COVID-19 immunization	IM	BLA; Priority Review	05/30/2025
elinzanetant	Bayer	Vasomotor symptoms associated with menopause	Oral	NDA	08/01/2025
telmisartan / amlodipine / indapamide	George Medicines	Hypertension (including initiation of treatment)	Oral	505(b)(2) NDA	08/06/2025
aceclidine	Lenz	Presbyopia	Ophthalmic	NDA	08/08/2025
cyclobenzaprine	Tonix	Fibromyalgia	SL	505(b)(2) NDA; Fast Track	08/15/2025
aficamten	Cytokinetics	Obstructive hypertrophic cardiomyopathy	Oral	NDA; Breakthrough Therapy, Orphan Drug	09/26/2025
sodium dichloroacetate (SL-1009)	Saol Therapeutics	Pyruvate dehydrogenase complex deficiency (PDCD)	Oral	NDA; Fast Track, Orphan Drug, RPD	12/03/2025
seasonal influenza/COVID-19 vaccine (mRNA-1083)	Moderna	COVID-19 immunization; Seasonal influenza immunization	IM	BLA; Fast Track	01/13/2026

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
brexpiprazole (Rexulti)	Otsuka	PTSD (in combination with sertraline)	Oral	sNDA	02/08/2025
epinephrine (Neffy)	ARS	Anaphylaxis (children weighing 15 to 30 kg)	Intranasal	sNDA; Fast Track, Priority Review	03/06/2025
cobicistat / darunavir (Prezcobix)	Janssen	HIV-1 infection (ages ≥ 6 years, weighing ≥ 25 kg)	Oral	sNDA	04/04/2025
roflumilast (Zoryve) 0.3% foam	Arcutis	PSO (scalp and body, ages ≥ 12 years)	Topical	sNDA	05/22/2025
meningococcal vaccine (Menquadfi)	Sanofi	Meningococcal immunization (ages 6 weeks to 23 months)	IM	sBLA	05/23/2025
tirzepatide (Zepbound)	Eli Lilly	Chronic HFpEF (with obesity)	SC	sNDA	Jul-Sep 2025
RSV vaccine (mRESVIA)	Moderna	RSV immunization (high-risk adults, ages 18-59 years)	IM	sBLA; Priority Review	07/11/2025
lumateperone (Caplyta)	Intra-Cellular Therapies	MDD (adjunct to antidepressants, adults)	Oral	sNDA	10/03/2025
roflumilast (Zoryve) 0.05% cream	Arcutis	Atopic dermatitis (ages 2 to 5 years)	Topical	sNDA	10/16/2025
finerenone (Kerendia)	Bayer	Chronic HF (LVEF ≥ 40% or preserved LVEF)	Oral	sNDA	11/10/2025

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
amisulpride	Sumitomo	Bipolar disorder	Oral	NDA	TBD
aroxybutynin / atomoxetine	Apnimed	OSA	Oral	NDA; Fast Track	TBD
baclofen / naltrexone / sorbitol	Pharnex	Charcot-Marie-Tooth disease	Oral	NDA; Fast Track, Orphan Drug	TBD
basimglurant	Noema	Trigeminal neuralgia	Oral	NDA; Fast Track	TBD
blarcamesine	Anavex	Alzheimer's disease	Oral	NDA	TBD
brilaroxazine	Reviva	Schizophrenia	Oral	NDA	TBD
cagrilintide / semaglutide	Novo Nordisk	Obesity; T2DM	SC	NDA	TBD
camlipixant	GlaxoSmithKline	Chronic cough	Oral	NDA	TBD
carbachol / brimonidine	Visus	Presbyopia	Ophthalmic	505(b)(2) NDA	TBD
cytisinicline	Achieve	Smoking cessation	Oral	NDA; Breakthrough Therapy	TBD
d-cycloserine / lurasidone	Alvogen	Bipolar disorder (suicidal)	Oral	505(b)(2) NDA; Breakthrough Therapy, Fast Track	TBD
dipalmitoyl hydroxyproline (QRX003)	Quoin	Netherton syndrome	Topical	NDA	TBD
epinephrine SL	Aquestive	Anaphylaxis	SL	NDA	505(b)(2) NDA; Fast Track
esreboxetine	Axsome / Pfizer	Fibromyalgia	Oral	NDA	TBD
gepotidacin	GlaxoSmithKline	Urogenital gonorrhea	Oral	NDA; QIDP	TBD
Lyme disease vaccine	Valneva / Pfizer	Lyme disease immunization	IM	BLA; Fast Track	TBD
milsaperidone	Vanda	Bipolar disorder; schizophrenia	Oral	NDA	TBD
navacaprant	Neumora	MDD	Oral	NDA	TBD
obicetrapib	New Amsterdam	Dyslipidemia	Oral	NDA	TBD
orforglipron	Eli Lilly	Obesity; T2DM	Oral	NDA	TBD
oveporexton	Takeda	Narcolepsy	Oral	NDA; Breakthrough Therapy	TBD
paromomycin	Appili	Leishmaniasis	Topical	NDA; Orphan Drug	TBD
purified vero rabies vaccine (SP0087)	Sanofi	Rabies (post exposure treatment)	IM	BLA	TBD
quadrivalent influenza mRNA vaccine (mRNA-1010)	Moderna	Seasonal influenza vaccination	IM	BLA	TBD
ralinepag	United Therapeutics	PAH	Oral	NDA; Orphan Drug	TBD
retatrutide	Eli Lilly	Obesity; OSA; T2DM	SC	NDA	TBD
RSV live attenuated vaccine (SP0125)	Sanofi	RSV immunization	Intranasal	BLA	TBD
survodutide	Zealand / Boehringer Ingelheim	MASH; obesity; T2DM	SC	NDA	TBD
tavapadon	Abbvie	Parkinson's disease	Oral	NDA	TBD
tiratricol	Egetis	Monocarboxylate transporter 8 (MCT8) deficiency	Oral	NDA; Fast Track, Orphan Drug, RPD	TBD
tradipitant	Vanda / Eli Lilly	Emesis (motion sickness)	Oral	NDA	TBD
ulixacaltamide	Praxis	Essential tremor	Oral	NDA	TBD
valiltramiprosate	Alzheon	Alzheimer's disease	Oral	NDA; Fast Track	TBD
vidofludimus	Immunic	MS	Oral	NDA	TBD
zoliflodacin	Innoviva	Gonorrhea (uncomplicated)	Oral	NDA; Fast Track, QIDP	TBD

Name	Manufacturer	Clinical use	Dosage form	Development status	FDA decision
dextromethorphan / bupropion (Auvelity)	Axsome	Alzheimer’s disease-related neuropsychiatric symptoms	Oral	505b2 NDA; Breakthrough Therapy, Fast Track	TBD
finerenone (Kerendia)	Bayer	Chronic HFrEF	Oral	sNDA	TBD
semaglutide (NN9931)	Novo Nordisk	MASH	SC	sNDA; Breakthrough Therapy	TBD
semaglutide (Ozempic)	Novo Nordisk	Alzheimer's disease; diabetic retinopathy; PAD (with T2DM)	SC	NDA	TBD
semaglutide (Rybelsus)	Novo Nordisk	Obesity	Oral	sNDA	TBD
semaglutide (Wegovy)	Novo Nordisk	Chronic HFpEF; osteoarthritis (with obesity)	Oral	SC	sNDA

Name	Manufacturer	Clinical use	Dosage form	Development status
amivantamab / hyaluronidase (Rybrevant SC)	Janssen	NSCLC	SC infusion	CRL
garadacimab	CSL	HAE	SC	CRL
glepaglutide	Zealand	Short bowel syndrome (with intestinal failure)	SC	CRL
govorestat	Applied Therapeutics	Classic galactosemia	Oral	CRL
sotagliflozin (Inpefa)	Lexicon	T1DM (with CKD)	Oral	CRL
tabelecleucel	Pierre Fabre / Atara	Epstein-Barr virus-associated post-transplant lymphoproliferative disease	IV	CRL

5-FU 5-Fluorouracil
6MWD 6 Minute Walking Distance
6MWT 6 Minute Walking Test
ABSSSI Acute Bacterial Skin and Skin Structure Infection
ACC American College of Cardiology
ACEI Angiotensin-Converting Enzyme Inhibitor
AChR Acetylcholine Receptor
ACR20 American College of Rheumatology 20% Improvement
ACR50 American College of Rheumatology 50% Improvement
ACR70 American College of Rheumatology 70% Improvement
ADC Antibody-Drug Conjugate
ADHD Attention Deficit Hyperactivity Disorder
ADL Activities of Daily Living
ALK Anaplastic Lymphoma Kinase
ALK+ Anaplastic Lymphoma Kinase-positive
ALL Acute Lymphoblastic Leukemia
ALS Amyotrophic Lateral Sclerosis
ALSFRS-R Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised
ALT Alanine Transaminase
AMD Age-Related Macular Degeneration
AML Acute Myeloid Leukemia
ANCA Antineutrophil Cytoplasmic Antibodies
APOE4 Apolipoprotein E4 gene variant
ARB Angiotensin II Receptor Blocker
ARNI Angiotensin Receptor II Blocker – Neprilysin Inhibitor
AS Ankylosing Spondylitis
ASCVD Atherosclerotic Cardiovascular Disease
AST Aspartate Aminotransferase
BCG Bacillus Calmette-Guérin
BCVA Best Corrected Visual Acuity
BLA Biologics License Application
BMI Body Mass Index
BMT Bone Marrow Transplant
BP Blood Pressure
BPH Benign Prostatic Hyperplasia
BRAF V-Raf Murine Sarcoma Viral Oncogene Homolog B1
BTK Bruton’s Tyrosine Kinase
BSA Body Surface Area
BsUFA Biosimilar User Fee Act
CABP Community Acquired Bacterial Pneumonia
CAP Community Acquired Pneumonia
CAR T Chimeric Antigen Receptor T-Cell
CD Crohn's Disease
CD3 Cluster of Differentiate 3
CD19 Cluster of Differentiate 19
CD20 Cluster of Differentiate 20
CD38 Cluster of Differentiate 38
CD79b Cluster of Differentiate 79b
CDC Centers for Disease Control and Prevention
CF Cystic Fibrosis
CFTR Cystic Fibrosis Transmembrane Conductance Regulator
CHF Congestive Heart Failure
CI Confidence Interval
CKD Chronic Kidney Disease
CLDN18.2+ Claudin-18.2-positive
CLL Chronic Lymphocytic Leukemia
CML Chronic Myeloid Leukemia
CMS Centers for Medicare & Medicaid Services
CNS Central Nervous System
COPD Chronic Obstructive Pulmonary Disease
COVID-19 Coronavirus Disease 2019
CRC Colorectal Cancer
CRL Complete Response Letter
CRR Complete Response Rate
CRS Cytokine Release Syndrome
CSF Colony Stimulating Factor
CTLA-4 Cytotoxic T-Lymphocyte-Associated Protein 4
CV Cardiovascular
CVD Cardiovascular Disease
CYP3A4 Cytochrome P-450 3A4
CYP450 Cytochrome P-450
DAS28-CRP Disease Activity Score-28 with C Reactive Protein
DBP Diastolic Blood Pressure
DCR Disease Control Rate
DEA Drug Enforcement Administration
DED Dry Eye Disease
DLBCL Diffuse Large B Cell Lymphoma
DMARD Disease Modifying Antirheumatic Drug
DMD Duchenne Muscular Dystrophy
DME Diabetic Macular Edema
dMMR DNA mismatch repair
DMT Disease Modifying Therapy
DNA Deoxyribonucleic Acid
DOR Duration of Response
DPP-4 Dipeptidyl Peptidase 4
DR Delayed-Release
DSM-5 Diagnostic and Statistical Manual of Mental Disorders, 5th edition
EASI-75 Eczema Area and Severity Index ≥ 75% Reduction
ECOG Eastern Cooperative Oncology Group
EDSS Expanded Disability Status Scale
eGFR estimated Glomerular Filtration Rate
EGFR Epidermal Growth Factor Receptor
ER Extended-Release
ERA Endothelin Receptor Agonist
ERK Extracellular signal-Regulated Kinase
ESA Erythropoietin Stimulating Agent
ESRD End-Stage Renal Disease
EUA Emergency Use Authorization
FDA Food and Drug Administration
FEV₁ Force Expiratory Volume in 1 Second
FH Familial Hypercholesterolemia
FLT3 FMS-Like Tyrosine Kinase-3
FMS Feline McDonough Sarcoma
FVC Forced Vital Capacity
GABA-A Gamma-Aminobutyric Acid Receptor Type A
G-CSF Granulocyte Colony Stimulating Factor
GERD Gastroesophageal Reflux Disease
GGT Gamma-Glutamyl Transferase
GI Gastrointestinal
GIST Gastrointestinal Stromal Tumor
GLP-1RA Glucagon-Like Peptide-1 Receptor Agonist
GM-CSF Granulocyte-Macrophage Colony Stimulating Factor
GVHD Graft Versus Host Disease
H Half
H3 K27M Histone 3 Lysine 27-to-Methionine
HAE Hereditary Angioedema
HAM-A Hamilton Anxiety Rating Scale
HAM-D Hamilton Depression Rating Scale
HAMD-17 Hamilton Depression Rating Scale
HAP Healthcare-Associated Pneumonia
Hb Hemoglobin
HbA1c Hemoglobin A1c
HBV Hepatitis B Virus
HCC Hepatocellular Carcinoma
HCP Healthcare Professional
HCV Hepatitis C Virus
HDRS-17 Hamilton Depression Rating Scale
HeFH Heterozygous Familial Hypercholesterolemia
HER Human Epidermal Growth Factor Receptor
HER2 Human Epidermal Growth Factor Receptor 2
HER2- Human Epidermal Growth Factor Receptor 2-negative
HER2+ Human Epidermal Growth Factor Receptor 2-positive
HF Heart Failure
HFA Hydrofluoroalkane
HFpEF Heart Failure with preserved Ejection Fraction
HFrEF Heart Failure with reduced Ejection Fraction
HIT Heparin Induced Thrombocytopenia
HIV Human Immunodeficiency Virus
HIV-1 Human Immunodeficiency Virus-1
HR Hormone Receptor
HR- Hormone Receptor-negative
HR+ Hormone Receptor-positive
HoFH Homozygous Familial Hypercholesterolemia
HS Hidradenitis Suppurativa
HSCT Hematopoietic Stem Cell Transplant
HSV Herpes Simplex Virus
HTN Hypertension
IBS Irritable Bowel Syndrome
IBS-C Irritable Bowel Syndrome, Constipation Predominant
ICER Institute for Clinical and Economic Review
ICS Inhaled Corticosteroid
IDH Isocitrate Dehydrogenase
IGA Investigator's Global Assessment
IgA Immunoglobulin A
IgG Immunoglobulin G
IgG1 Immunoglobulin G1
IgG4 Immunoglobulin G4
IHC Immunohistochemistry
IL-4 Interleukin-4
IL-5 Interleukin-5
IL-8 Interleukin-8
IL-12 Interleukin-12
IL-13 Interleukin-13
IL-17 Interleukin-17
IL-23 Interleukin-23
IL-31 Interleukin-31
IM Intramuscular
IR Immediate-Release
IRB Institutional Review Board
ISH In Situ Hybridization
ITP Immune Thrombocytopenic Purpura
ITT Intent-To-Treat
IV Intravenous
JAK Janus Kinase Inhibitor
JIA Juvenile Idiopathic Arthritis
KIT c-KIT Proto-Oncogene
KMT2A Lysine (K)-Specific Methyltransferase 2A
KRAS Kirsten Rat Sarcoma viral oncogene homolog
LABA Long-Acting Beta Agonist
LAMA Long-Acting Muscarinic Antagonist
LDL-C Low-Density Lipoprotein Cholesterol
LPAD Limited Population Pathway for Antibacterial and Antifungal Drugs
LRP4 Low density Lipoprotein-Related Protein 4
LSM Least Square Mean
LVEF Left Ventricular Ejection Fraction
mAb Monoclonal Antibody
MACE Major Adverse Cardiovascular Events
MADRS Montgomery – Åsberg Depression Rating Scale
MAPK Mitogen-Activated Protein Kinase
MASH Metabolic Dysfunction-Associated Steatohepatitis
MDD Major Depressive Disorder
MDI Metered Dose Inhaler
MDR Multi-Drug Resistant
MECP2 Methyl-CpG Binding Protein 2
MEK Mitogen-Activated Extracellular Signal-Regulated Kinase
MET Mesenchymal Epithelial Transition factor receptor
MI Myocardial Infarction
mITT modified Intent-To-Treat
MRI Magnetic Resonance Imaging
mRNA messenger Ribonucleic Acid
MRSA Methicillin-Resistant Staphylococcus Aureus
MS Multiple Sclerosis
MSI-H Microsatellite Instability-High
mTOR mechanistic Target of Rapamycin
MuSK Muscle-Specific tyrosine Kinase
N/A Not Applicable
NAFLD Nonalcoholic Fatty Liver Disease
NASH Non-Alcoholic Steatohepatitis
NCCN National Comprehensive Cancer Network
NCT National Clinical Trials
NDA New Drug Application
NHL Non-Hodg
NIH National Institutes of Health
nr-axSpA Non-Radiographic Axial Spondyloarthritis
NRAS Neuroblastoma RAS Proto-Oncogene
NRG1+ Neuregulin-1-positive
NSAID Non-Steroidal Anti-Inflammatory Drug
NSCLC Non-Small Cell Lung Cancer
NTRK Neurotrophic Tyrosine Receptor Kinase
NYHA New York Heart Association
ODT Orally Disintegrating Tablet
OR Odds Ratio
ORR Objective Response Rate
OS Overall Survival
OSA Obstructive Sleep Apnea
OTC Over-the-Counter
PAD Peripheral Arterial Disease
PAH Pulmonary Arterial Hypertension
PARP Poly (ADP-Ribose) Polymerase
PAS Prior Approval Supplement
PASI Psoriasis Area and Severity Index
PASI 50 Psoriasis Area and Severity Index 50% Reduction
PASI 75 Psoriasis Area and Severity Index 75% Reduction
PASI 90 Psoriasis Area and Severity Index 90% Reduction
PASI 100 Psoriasis Area and Severity Index 100% Reduction
PCI Percutaneous Coronary Intervention
PCSK9 Proprotein Convertase Subtilisin Kexin 9
PD-1 Programmed Death Protein 1
PD-L1 Programmed Death-Ligand 1
PDE3 Phosphodiesterase 3
PDE4 Phosphodiesterase 4
PDE5 Phosphodiesterase 5
PDUFA Prescription Drug User Fee Application
PFS Progression-Free Survival
PGA Physician Global Assessment
PHI Primary Humoral Immunodeficiency
PI3K Phosphatidylinositol-3-kinase
PNH Paroxysmal Nocturnal Hemoglobinuria
PsA Psoriatic Arthritis
PSO Plaque Psoriasis
PTCA Percutaneous Transluminal Coronary Angioplasty
PTSD Post-Traumatic Stress Disorder
Q Quarter
QIDP Qualified Infectious Diseases Product
QOL Quality of Life
R/R Relapsed or Refractory
R-CHOP Rituximab, Cyclophosphamide, Doxorubicin, Vincristine, Prednisone
RA Rheumatoid Arthritis
RAF Rapidly Accelerated Fibrosarcoma
RARA Retinoic Acid Receptor alpha
RAS Ras Protein Superfamily
RBC Red Blood Cell
RCC Renal Cell Carcinoma
REMS Risk Evaluation and Mitigation Strategy
RMAT Regenerative Medicine Advanced Therapy
RNA Ribonucleic Acid
ROS1 ROS Proto-Oncogene 1
RPD Rare Pediatric Disease
RRR Relative Risk Reduction
RSV Respiratory Syncytial Virus
RTOR Real-Time Oncology Review
RVO Retinal Vein Occlusion
SARS-CoV-2 Severe Acute Respiratory Syndrome-Associated Coronavirus-2
sBLA supplemental Biologics License Application
SBP Systolic Blood Pressure
SC Subcutaneous
SCCHN Squamous Cell Cancer of the Head and Neck
SCD Sickle Cell Disease
SCLC Small Cell Lung Cancer
SCT Stem Cell Transplant
SGLT2 Sodium-Glucose Co-Transporter 2
SL Sublingual
SLE Systemic Lupus Erythematosus
SLL Small Lymphocytic Lymphoma
sNDA supplemental New Drug Application
SNRI Serotonin and Norepinephrine Reuptake Inhibitor
SOC Standard of Care
SOD-1 Superoxide Dismutase - Type 1
sPGA static Physician Global Assessment
SR Sustained-Release
SSRI Selective Serotonin Reuptake Inhibitor
SSSI Skin and Skin Structure Infection
T1DM Type 1 Diabetes Mellitus
T2DM Type 2 Diabetes Mellitus
TBD To Be Determined
TEAE Treatment-Emergent Adverse Event
TKI Tyrosine Kinase Inhibitor
TNBC Triple Negative Breast Cancer
TNF Tumor Necrosis Factor
TNFα Tumor Necrosis Factor-alpha
UA Unstable Angina
UC Ulcerative Colitis
ULN Upper Limit of Normal
U.S. United States
UTI Urinary Tract Infection
VAS Visual Analog Scale
VEGF Vascular Endothelial Growth Factor
VTE Venous Thromboembolism
WBC White Blood Cell
WHO World Health Organization
XR Extended-Release

Quarterly Drug Pipeline: January 2025 - Prime Therapeutics

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Quarterly Drug Pipeline: January 2025

Editor-in-chief's message

Maryam Tabatabai Associate Vice President, Clinical Information

Editorial team

Maryam Tabatabai, PharmD

Consultant panel

Deep dive

ataluren (Translarna) oral

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

avutometinib + defactinib oral

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

clesrovimab IM

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

condoliase intervertebral disc injection

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

etripamil (Cardamyst) intranasal

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

fitusiran SC

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

pegzilarginase IV, SC

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

sebetralstat oral

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Financial forecast (reported in millions)

sepiapterin oral

Proposed indications

Clinical overview

Place in therapy

FDA approval timeline

Maryam Tabatabai
Associate Vice President, Clinical Information