Quarterly Drug Pipeline: October 2024

Clinical insights and competitive intelligence on anticipated drugs in development

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 U.S. Food and Drug Administration (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

With only a couple of months left in 2024, thus far the agency has approved 37 novel drugs, compared to about 43 novel approvals about the same time last year. For the remainder of the year, 22 notable drugs filed with the agency are profiled, each with an anticipated FDA decision in 2024.

Some of the recent noteworthy approvals include the first engineered cell therapy for unresectable or metastatic synovial sarcoma, new mechanisms of action for gastrointestinal cancers and schizophrenia, first SC formulation of a PDL-1 inhibitor for select cancers, new options for the neurological manifestations of Niemann-Pick disease – a rare progressive genetic disorder, two Accelerated Approvals for primary biliary cholangitis – a rare liver disease, a SC formulation of ocrelizumab (Ocrevus) for multiple sclerosis (MS), the first nasal formulation of epinephrine and a self/caregiver-administered option for intranasal flu vaccine.

While numbers do not tell the entire story, they do represent significant innovation in patient care and advance public health for the American public.

On the horizon

The FDA decisions for specialty medications (75%) and for Orphan Drugs (35%) continue to grow for agents with applications submitted to the FDA. Four agents submitted to the FDA are seeking FDA’s Accelerated Approval, which allows for earlier drug approval for serious conditions that fill an unmet need based on a surrogate endpoint reasonably likely to predict a clinical benefit.

Notable FDA decisions expected for fourth quarter 2024 include:

New indication for tirzepatide (Zepbound) for moderate-to-severe obstructive sleep apnea and obesity
Accelerated Approvals for two Orphan Drugs – a one-time gene therapy for an ultrarare protein deficiency and a drug for biliary tract cancer
CAR T cell therapy for bone marrow cancer
Several agents for rare diseases
o First-in-class encapsulated cell therapy implant for a neurodegenerative ophthalmic disorder
o Oral option for classic congenital adrenal hyperplasia, related to an enzyme deficiency
o Injectable for familial chylomicronemia syndrome, characterized by severe hypertriglyceridemia
o Injectable for short bowel syndrome with intestinal failure

We hope you enjoy the report!

Maryam Tabatabai
Vice President, Clinical Information

Editorial team

Maryam Tabatabai, PharmD

Editor-In-Chief
Vice President, Clinical Information

Carole Kerzic, RPh
Executive Editor
Clinical Pharmacist, Drug Information

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

Consultant panel

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

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

Katie Lockhart
Director, Trend and Forecasting Analytics

Olivia Pane, PharmD, CDCES
Clinical Pharmacist, Drug Information

Devon Trumbower, PharmD, BCPS
Clinical Pharmacist

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.

All brand names are property of their respective owners.

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 pink throughout the publication.

olezarsen SC

Manufacturer: Akcea/Ionis

Proposed indications

Familial chylomicronemia syndrome (FCS)

Clinical overview

Mechanism of action

Olezarsen is an RNA-targeted ligand conjugated antisense (LICA) agent that lowers plasma triglyceride (TG) levels by reducing hepatic synthesis of apolipoprotein C-III (apoC-III).

Clinical trials

The international, double-blind, placebo-controlled, phase 3 BALANCE trial (NCT04568434) evaluated olezarsen in 66 adults with genetically identified FCS. Enrolled patients had a baseline fasting TG level of ≥ 880 mg/dL (9.9 mmol/L) and were instructed to follow a diet containing ≤ 20 g of total fat per day. The trial had two primary endpoints which were the change in fasting TG levels from baseline to six months with olezarsen 80 mg compared to placebo and with olezarsen 50 mg compared to placebo. The study reported a statistically significant reduction in fasting TG levels from baseline with olezarsen 80 mg compared to placebo at six months (LS mean difference, -43.5 percentage points; p<0.001), which persisted at 12 months. The olezarsen 50 mg dose did not result in a significant reduction in fasting TG levels from baseline compared to placebo at six months (LS mean difference, -22.4 percentage points; p=0.08), but a greater reduction was seen at 12 months (LS mean change, -43.8 percentage points). As secondary endpoints, olezarsen 80 mg was associated with a lowering of apoC-III levels from baseline compared to six and 12 months (LS mean difference, -73.7 and -81.3 percentage points, respectively) compared to placebo. ApoC-III lowering was also seen with olezarsen 50 mg from baseline at six and 12 months (LS mean difference, -65.5 and -77.1 percentage points, respectively) compared to placebo. During the 12-month time period, a lower incidence of acute pancreatitis was reported with olezarsen; one episode each was reported with 80 mg and 50 mg doses compared to 11 episodes with placebo. Olezarsen was generally well tolerated. TEAEs of moderate severity reported with olezarsen included chills, myalgia, trismus, chest discomfort, diarrhea, flushing, vomiting, alopecia and transient platelet decrease.

Dosage and administration

In the clinical trial, olezarsen was administered at a dose of 80 mg or 50 mg SC every four weeks for 49 weeks.

Place in therapy

FCS is a rare, inherited condition characterized by severe hypertriglyceridemia and is estimated to affect one to 13 individuals per 1,000,000. The majority of FCS cases are caused by gene mutations resulting in a lack of a functional lipoprotein lipase (LPL) protein preventing the body from breaking down dietary and endogenous TGs. This leads to the abnormal persistent circulation of chylomicrons and very high triglycerides in the blood that can accumulate in various organs. The most serious complication of FCS acute pancreatitis, which can lead to pancreatic damage or diabetes, and can be life-threatening. Other signs and symptoms of FCS include abdominal pain, nausea, fatigue, diarrhea, hepatosplenomegaly, eruptive xanthomas, lipemia retinalis, and failure to thrive.

Standard lipid-lowering medications (e.g., statins, fibrates, niacin) and plasmapheresis are not effective in patients with FCS. The SOC for FCS is a fat restricted diet to < 10% of calories or < 20 grams per day. Patients with FCS should also maintain a healthy weight, exercise, and avoid processed foods, alcohol and smoking. If approved, olezarsen would be the first agent available in the U.S. to treat FCS. Notably, the antisense oligoneucleotide volanosorsen, which targets apoC-III messenger RNA, had demonstrated reduced plasma levels of apoC-III in clinical trials; however, it did not gain FDA approval due to an increased risk of thrombocytopenia. The inclusion of a triantennary N-acetylgalactosamine (GalNAc3) ligand in olezarsen’s molecular structure may mitigate the risk of thrombocytopenia. Thrombocytopenia was not reported in the published BALANCE study for olezarsen.

Olezarsen is also being evaluated in a phase 3 trial for the treatment of severe hypertriglyceridemia in combination with optimized lipid lower agents. In addition, the RNA interference (RNAi) agent, plozasiran is in phase 3 trials for FCS. Arrowhead Pharmaceuticals plans to submit plozasiran to the FDA in late 2024.

FDA approval timeline

Dec. 19, 2024

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

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales	$0	$22	$25	$42	$73

glepaglutide SC

Manufacturer: Zealand

Proposed indications

Short bowel syndrome (SBS) in adults with intestinal failure

Clinical overview

Mechanism of action

Glepaglutide is a long‐acting glucagon‐like peptide‐2 (GLP-2) analog, a class of drugs that have been shown to stimulate proximal intestinal blood flow, increase splanchnic oxygenation and angiogenesis, and promote intestinal mucosal growth.

Clinical trials

The double-blind, placebo-controlled, phase 3 EASE-1 trial (NCT03690206) evaluated glepaglutide in 106 adults with SBS with intestinal failure who required parenteral support (PS) at least three days per week. At 24 weeks, total weekly volume of PS (primary endpoint) was reduced from baseline by 5.13 liters with twice-weekly doses of glepaglutide (p=0.0039) and 3.13 liters with once-weekly doses (not statistically significant), compared to 2.85 liters with placebo. At weeks 20 and 24, clinical response, defined as ≥ 20% reduction in weekly PS volume, was achieved in 65.7% (p=0.0243) of patients who received glepaglutide twice weekly, 45.7% who received glepaglutide once weekly, and 38.9% who received placebo. In addition, 14% of patients in the glepaglutide twice-weekly group became independent of PS compared to no patients in the placebo group. Glepaglutide was well tolerated. The most common TEAEs with glepaglutide were injection site reactions and GI events.

The EASE-2 and EASE-3 long-term extension trials of glepaglutide in patients with SBS are ongoing. An interim analysis of EASE-2 conducted at six months showed that key efficacy endpoints were maintained or continued to improve with glepaglutide beyond the initial 24 weeks of treatment. Additional patients in the glepaglutide once- and twice-weekly dosing groups were able to wean off PS. The safety profile was like that reported in the EASE-1 trial.

Dosage and administration

In the EASE-1 trial, patients were randomized to glepaglutide 10 mg doses administered via SC injection once or twice a week.

Place in therapy

SBS is a rare, chronic malabsorptive disorder that occurs after extensive surgical resection of the small intestine for conditions such as Crohn’s disease, malignancy, trauma, radiation, or vascular insufficiency. Severe SBS‐associated intestinal failure (SBS‐IF) is considered when there is < 200 cm of functional small intestine remaining. Complications include severe diarrhea, fluid and electrolyte imbalances, vitamin deficiencies, renal stones, small intestinal bacterial overgrowth (SIBO), D-lactic acidosis as well as metabolic bone, liver, and biliary disease.

The management of patients with SBS-IF requires a comprehensive approach that may include long-term use of parenteral nutrition (PN) if nutritional needs are chronically not met via the enteral route alone. However, PN carries significant risks (e.g., IV catheter infection), and inhibits improved functioning of the remaining bowel, also known as intestinal adaptation.

GLP-2 plays an important role in intestinal adaptation, which is a natural process that occurs during the first two years after extensive intestinal resection. During this time, structural and functional changes occur in the remaining intestine to improve nutrient and fluid absorption. The GLP-2 analog teduglutide (Gattex) and the recombinant growth hormone somatropin (Serostim) are FDA-approved for SBS. Both enhance fluid and nutrient absorption and decrease PN requirements in patients with SBS. If approved, glepaglutide will compete directly with Gattex in adults. While Gattex is indicated for use in patients as young as 1 year of age, glepaglutide has only been studied in adults. In non-comparison trials, once or twice weekly glepaglutide and once daily Gattex resulted in a similar reduction in weekly PS volume (approximately 2-liter decrease in PS when each were compared to placebo). Gattex is available as an autoinjector for self- or caregiver-administration. The same is anticipated with glepaglutide with the benefit of a reduced injection burden.

The GLP-2 analog apraglutide is also in phase 3 trials for SBS with once-weekly SC dosing. Ironwood is expected to submit apraglutide to the FDA in the first quarter of 2025.

FDA approval timeline

Dec. 20, 2024

FDA designations: Orphan Drug

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales	$0	$11	$41	$82	$129

tirzepatide (Zepbound) SC

Manufacturer: Eli Lilly

Proposed indications

Moderate-to-severe obstructive sleep apnea (OSA) and obesity

Clinical overview

Mechanism of action

Eladocagene exuparvovec is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy. It delivers a functional dopa decarboxylase (DDC) gene to increase AADC enzyme levels and restore dopamine production.

Clinical trials

Two randomized, double-blind, phase 3, SURMOUNT-OSA trials (NCT05412004) evaluated Zepbound for the treatment of moderate-to-severe OSA (defined as ≥ 15 apnea-hypopnea index [AHI] events/hour) in a total of 469 adults with obesity (defined as BMI ≥ 30 kg/m²), without (Trial 1) and with (Trial 2) positive airway pressure (PAP) therapy. Enrolled patients did not have diabetes. All study participants received regular counseling sessions regarding the maintenance of healthy nutrition while adhering to a 500 kcal/day deficit and ≥ 150 minutes/week of physical activity. At baseline, in Trials 1 and 2, the mean AHI was 51.5 and 49.5 events/hour, respectively, and the mean BMI was 39.1 kg/m² and 38.7 kg/m², respectively. At week 52, the estimated difference with Zepbound compared to placebo in the primary endpoint of change in AHI from baseline was -20 events/hour (p<0.001) in Trial 1 and -23.8 events/hour in Trial 2 (p<0.001). Trials 1 and 2 also reported a significant reduction in body weight with Zepbound compared to placebo (difference in percent change: -16.1% in Trial 1 and -17.3% in Trial 2), a key secondary endpoint. Zepbound also led to improvements in hypoxic burden, high-sensitivity C-reactive protein (hsCRP) concentration, systolic blood pressure and patient-reported sleep-related outcomes. The most common TEAEs were mild to moderate GI symptoms.

Dosage and administration

In the clinical trials, Zepbound was self-administered SC using a single-dose pen autoinjector once weekly for 52 weeks. The starting dose was 2.5 mg and was increased by 2.5 mg every four 4 weeks to a maximum of 10 mg or 15 mg.

Place in therapy

OSA is caused by the collapse of soft tissue in the back of throat blocking the upper airway. This results in partial reductions (hypopneas) and complete pauses (apneas) in breathing that last from seconds to minutes and a decrease in oxygen saturation and/or waking from sleep. Notably, OSA is an independent risk factor for CVD. Approximately $30 million adults in the U.S. have OSA, and an estimated 80% of cases remain undiagnosed. Incidence of OSA is higher in men (23%) than women (9%). Higher weight increases the likelihood of airway obstruction during sleep and approximately 70% of patients with OSA also have obesity; in addition, an estimated 40% of people with obesity have OSA.

Weight loss can help manage OSA, with improvement or resolution of OSA reported with 5% to 10% reduction in weight. Continuous positive airway pressure (CPAP) that keeps the airway open during sleep is commonly used in patients with moderate to severe OSA. However, it is estimated that about 30% to 50% of people prescribed CPAP will discontinue use within the first year. Oral appliances may also be effective for mild to moderate cases. In addition, surgical procedures (e.g., uvulopalatopharyngoplasty, adenotonsillectomy) may be performed to correct an anatomical deformity if the above noninvasive treatments are unsuccessful.

While the pharmaceutical agent, solriamfetol (Sunosi), is indicated to improve wakefulness in adults with OSA, it does not improve apnea and hypopnea that is associated with OSA. If approved, Zepbound will be the first medication to address this unmet need in patients with OSA. While weight loss may be a major factor for OSA improvement seen with Zepbound, it is unclear if other mechanisms contribute to OSA improvement.

Moreover, Medicare and many private insurers do not cover the cost of medications indicated for weight management alone. However, the potential OSA indication for Zepbound could open the door for its coverage by Medicare. In March 2024, CMS issued a memo clarifying that anti-obesity medications (AOMs) that receive FDA approval for an additional medically accepted indication can be added to Medicare Part D plans, including Medicare Advantage, for that specific use. This also impacts coverage for semaglutide (Wegovy) which recently received a new CV indication. In addition, there is proposed legislation in Congress to remove the statutory prohibition on Medicare coverage of AOMs.

Zepbound and Wegovy are also in phase 3 trials for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), previously known as non-alcoholic steatohepatitis (NASH).

FDA approval timeline

Dec. 21, 2024 (if Priority Review granted)

FDA Designations: Fast Track

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales*	$0	$599	$1,233	$1,906	$2,454

^{*Projected sales reported for proposed OSA plus obesity indication}

crinecerfont oral

Manufacturer: Neurocrine

Proposed indications

Classic congenital adrenal hyperplasia (CAH)

Clinical overview

Mechanism of action

Crinecerfont is an oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist that decreases adrenocorticotropic hormone (ACTH) levels in the pituitary, leading to a decrease in the production of adrenal androgens.

Clinical trials

Crinecerfont was evaluated in a randomized, double-blind, placebo-controlled, phase 3 CAHtalyst trial (NCT04490915) in 182 adults with classic CAH on stable supraphysiologic doses of glucocorticoid (GC) therapy. Supraphysiologic doses are usually required to control androgen excess in classic CAH. Crinecerfont met the primary endpoint with a significant reduction from baseline in the daily GC dose, while maintaining androstenedione at a controlled level, compared to placebo at week 24 (change in GC dose, -27.3% versus -10.3%, respectively; p<0.001). Moreover, as a key secondary endpoint, 63% of patients in the crinecerfont group were able to receive a physiologic dose, versus a supraphysiologic dose, of GC compared to 18% in the placebo group (p<0.001). In addition, at week four, the androstenedione level had decreased in the crinecerfont group but increased in the placebo group (p<0.001), another key secondary endpoint. The most common adverse effects were fatigue and headache which was reported with crinecerfont and placebo.

Crinecerfont was also evaluated in a multinational, randomized, double-blind, placebo-controlled, phase 3 CAHtalyst Pediatric trial (NCT04806451) that enrolled 103 pediatric patients with classic CAH on stable supraphysiologic doses of GC therapy. Crinecerfont met the primary endpoint with a significant reduction from baseline in the androstenedione level compared to an increase with placebo (-197 ng/dL [-6.9 nmol/L] versus +71 ng/dL [+2.5 nmol/liter], respectively; p<0.001) at week four. In addition, while androstenedione control was maintained at week 28, there was a significant reduction in the mean GC dose (secondary endpoint) by 18% in the crinecerfont group compared to a 5.6% increase in the placebo group (p<0.001). The most common adverse events with crinecerfont were headache, pyrexia, and vomiting.

Dosage and administration

In the clinical trials, crinecerfont was administered orally twice daily. Adult patients received a total daily dose of 100 mg for 24 weeks. Pediatric patients received a dose of 25 mg, 50 mg, or 100 mg per day, depending on body weight, for 28 weeks.

Place in therapy

Classic CAH is a rare, genetic disorder that involves a deficiency of the 21-hydroxylase, an enzyme responsible for converting cholesterol to cortisol in the adrenal glands. A lack of this enzyme leads to a deficiency in cortisol and/or aldosterone and an excess of adrenal androgens. Classical CAH is diagnosed in infancy with mandatory neonatal screening in the U.S. Classic CAH is characterized by the development of atypical genitalia and salt-wasting, which could be life-threatening (adrenal crisis) if left untreated. In addition, excess adrenal androgens affect bone development and may cause rapid growth, early puberty, and infertility. The prevalence of classic CAH in the U.S. is about 1 in 10,000 to 1 in 23,000 people, with the highest reported among native Alaskans (1 in 280). In contrast, non-classic CAH is a more prevalent (1 in 100 to 1 in 1,000 individuals) and milder form of the disorder, with onset of symptoms in late childhood or early adulthood.

There is no cure for CAH. The goals for treatment of classic CAH are to prevent adrenal crisis and optimize growth, sexual maturation, and reproductive function. GC and mineralocorticoids are SOC for classic CAH. Mineralocorticoid agents are prescribed to replace aldosterone, and along with sodium supplementation help balance sodium and potassium levels in the body. GCs replace cortisol and control excess androgen production. However, high, or supraphysiologic, doses of GCs are often required to manage CAH symptoms, which can lead to short-term and long-term complications, such as growth and development issues in children, and weight gain or diabetes in adults.

Crinecerfont is a first-in-class CRF1 antagonist. If FDA-approved, it would be the ﬁrst non-GC therapy available in the U.S. for classic CAH, and it will be used as add-on to GC therapy. In clinical trials, the addition of crinecerfont to GC therapy resulted in a reduction in circulating androstenedione, a steroidal hormone involved in the production of estrogen and testosterone, and thereby allowed for a reduced GC dose to manage CAH symptoms.

A second CRF1 antagonist, tildacerfont by Eli Lilly, is being evaluated in phase 2 clinical trials for classic CAH and polycystic ovary syndrome (PCOS).

FDA approval timeline

Dec. 29, 2024 (capsule) and Dec. 30, 2024 (oral solution)

FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales	$0	$82	$177	$258	$311

suzetrigine oral

Manufacturer: Vertex

Proposed indications

Moderate to severe acute pain

Clinical overview

Mechanism of action

Suzetrigine is a non-opioid, oral, selective NaV1.8 pain signal inhibitor. NaV1.8 is a voltage-gated sodium channel that transmits pain signals in peripheral nociceptors.

Clinical trials

Suzetrigine was evaluated in two randomized, double-blind, placebo-controlled, phase 3 trials in adults experiencing moderate to severe acute pain following abdominoplasty (NCT05558410; n=1,118) or bunionectomy (NCT05553366; n=1,073). Interim data in both studies revealed that suzetrigine led to a significant improvement in the primary endpoint of the time-weighted sum of the pain intensity difference from 0 to 48 hours post-surgery (SPID48) compared to placebo (LS mean difference in SPID48, 48.4 [p<0.0001] post-abdominoplasty and 29.3 [p=0.0002] post-bunionectomy). The time to onset of a meaningful relief in pain, defined as ≥ 2-point reduction on the Numeric Pain Rating Scale (NPRS), was more rapid with suzetrigine compared to placebo (median, 2 hours versus 8 hours, respectively, post abdominoplasty [p<0.0001]; median, 4 hours versus 8 hours, respectively, post bunionectomy [p<0.0016]). When testing superiority of suzetrigine over hydrocodone bitartrate/acetaminophen (HB/APAP) as a key secondary endpoint, neither study demonstrated superiority of suzetrigine over HB/APAP based on SPID48 (LS mean difference in SPID48, 6.6 [p=0.2781] post-abdominoplasty and -20.2 [p=0.0016] post-bunionectomy). In general, suzetrigine was well tolerated.

A single-arm, phase 3 trial (NCT05661734) evaluated suzetrigine for the treatment of moderate to severe pain in a broad range of surgical and non-surgical adult patients. According to the patient global assessment (PGA), 83.2% of patients reported that suzetrigine’s effectiveness in treating pain was good, very good or excellent. In general, suzetrigine was well tolerated.

Dosage and administration

In clinical trials, suzetrigine was administered orally with an initial dose of 100 mg followed by 50 mg every 12 hours for up to 14 days. Hydrocodone bitartrate/acetaminophen was administered as 5 mg/325 mg orally every 6 hours over 42 hours.

Place in therapy

According to the CDC, acute pain usually has a sudden onset, duration of less than one month and is often caused by injury, trauma, or medical treatments (e.g., surgery). However, unresolved acute pain, lasting up to three months, can progress into chronic pain.

The CDC recommends that patients receive treatment for pain that provides the greatest benefits relative to its risks. Use of nonpharmacologic and nonopioid pharmacologic therapies should be maximized as appropriate. Opioids have a key role for acute pain associated with severe traumatic injuries, invasive surgical procedures associated with moderate to severe post-operative pain, and other severe acute pain when NSAIDs and other therapies are contraindicated or likely to be ineffective. When use of an opioid is warranted, an immediate-release agent should be prescribed at the lowest effective dose and for no longer than the expected duration of pain severe enough to require an opioid. Notably, all immediate-release opioid pain medications are associated with serious risks of misuse, abuse, addiction, overdose, and death. In addition, prescribers should consider concurrent medical conditions, including sleep apnea, pregnancy, renal or hepatic insufficiency, mental health conditions and substance use disorders, when assessing risks of opioid therapy.

Suzetrigine is a non-opioid, first-in-class NaV1.8 pain signal inhibitor. Unlike opioid medications, it works at peripheral pain receptors and, therefore, is not associated with CNS adverse effects. In clinical trials, suzetrigine was safe and effective in treating adults with moderate to severe acute pain in post-surgical and non-surgical settings. However, superiority compared to hydrocodone bitartrate/acetaminophen was not demonstrated when evaluated in post-surgical patients. If approved, suzetrigine will provide the first new mechanism of action for the short-term (up to 14 days) treatment of acute pain in about 20 years.

Suzetrigine is also in phase 2 trials for the treatment of neuropathic pain associated with diabetes and lumbosacral radiculopathy.

FDA approval timeline

Jan. 30, 2025

FDA designations: Breakthrough Therapy, Fast Track, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales	$0	$189	$395	$586	$734

datopotamab deruxtecan IV

Manufacturer: Daiichi Sankyo/AstraZeneca

Proposed indications

Second-line treatment of locally advanced or metastatic, nonsquamous non-small cell lung cancer (NSCLC)
Unresectable or metastatic, HR-positive/HER2-negative, breast cancer (immunohistochemistry [IHC] 0, IHC 1+ or IHC 2+/in situ hybridization-negative [ISH-]) after prior systemic therapy

Clinical overview

Mechanism of action

Datopotamab deruxtecan (dato-DXd) is an engineered trophoblast cell-surface antigen 2 (TROP2)-directed antibody drug conjugate (ADC). It contains a humanized anti-TROP2 IgG1 monoclonal antibody attached to topoisomerase I inhibitor (DXd) payload. Dato-DXd binds to TROP2 on tumor cells and is internalized into the tumor cell where the cytotoxic payload is released resulting in tumor cell death.

Clinical trials

NSCLC

The randomized, global, open-label, phase 3 TROPION-Lung01 trial (NCT04656652) evaluated dato-DXd in adults with locally advanced or metastatic NSCLC (with and without actionable genomic alterations) who were treated with at least one prior therapy, including an approved targeted therapy and platinum-based chemotherapy. The median treatment duration was 4.2 months with dato-DXd and 2.8 months with docetaxel. Information presented here will focus on data in patients with nonsquamous NSCLC (n=468). Dual primary endpoints were PFS and OS. In the primary analysis, dato-DXd reduced the risk of disease progression or death by 37% compared to docetaxel (median PFS, 5.5 months versus 3.6 months, respectively; hazard ratio, 0.63). Regarding secondary endpoint results with dato-DXd and docetaxel (respectively), ORR was 31.2% and 12.8%, and the median DOR was 7.7 months and 5.6 months. The final analysis reported, after a median follow-up of 23.1 months, the median OS was numerically longer with dato-DXd compared to docetaxel (14.6 months versus 12.3 months, respectively; hazard ratio, 0.84 [95% CI, 0.68 to 1.05]), but the difference was not statistically significant. Overall, grade ≥ 3 TEAEs were reported in fewer patients who received dato-DXd (25.6%) compared to those who were given docetaxel (42.1%), including neutropenia (0.7% versus 23.4%, respectively) and leukopenia (0% versus 13.1%, respectively). Although, grade ≥ 3 adjudicated drug-related interstitial lung disease or pneumonitis occurred more often with dato-DXd than docetaxel (3.7% versus 1.4%, respectively).

Breast Cancer

The ongoing, global, open-label, phase 3 TROPION-Breast01 trial (NCT05104866) evaluated dato-DXd in adults with inoperable or metastatic, HR-positive/HER2-negative breast cancer (HER2-negative defined as IHC 0, 1+ or 2+/ISH-) who had disease progression on endocrine therapy and had received one or two previous lines of chemotherapy; prior treatment with a TROP2-targeted therapy was not permitted. Dual primary endpoints were PFS and OS. A total of 732 patients were randomized to dato-DXd or investigator’s choice of chemotherapy (ICC). After a median duration of follow-up of 10.8 months, dato-DXd reduced the risk of progression or death compared with ICC by 37% (median PFS, 6.9 months versus 4.9 months, respectively; hazard ratio, 0.63; p<0.0001). Regarding secondary endpoint results with dato-DXd and ICC (respectively), ORR was 36.4% and 22.9%, and the median DOR was 6.7 months and 5.7 months. The overall rate of grade ≥ 3 TEAEs was lower with dato-DXd compared to ICC (20.8% versus 44.7%, respectively). Grade ≥ 3 stomatitis was reported more often with dato-DXd than ICC (6.4% versus 2.6%, respectively). Notably, grade ≥ 3 neutropenia occurred less often with dato-DXd compared to ICC (1.1% versus 30.8%, respectively).

Dosage and administration

In clinical trials, dato-DXd was administered via IV infusion at a dose of 6 mg/kg every three weeks until disease progression, unacceptable toxicity or other reasons. Patients were premedicated with antihistamines and acetaminophen, with or without glucocorticoids.

Place in therapy

It is estimated that 234,580 people in the U.S. will be diagnosed with lung cancer in 2024. Approximately 80% of lung cancer cases are classified as NSCLC, of which about 75% have nonsquamous histology. An estimated 313,510 new cases of breast cancer will occur in the U.S. in 2024. Approximately 90% of new breast cancer cases in women are HR-positive/HER2-negative.

Advanced cases of NSCLC are usually treated with chemotherapy, targeted agents and/or immunotherapy. For HR-positive/HER2-negative breast cancer, ADC therapies designed to deliver potent cytotoxic therapy directly to tumor cells are available and are preferred in the post-endocrine setting.

TROP2 is a cell surface glycoprotein that is overexpressed in many solid tumors, including NSCLC and breast cancer. It has been associated with drug resistance and has become a potential marker for poorer prognosis as well as a target for cancer therapy. The first TROP2-targeted ADC agent, sacituzumab govitencan (Trodelvy), was FDA-approved in 2020 and is a preferred second-line treatment for HR-positive/HER2-negative (HER2 IHC 1+ or 2+/ISH negative) breast cancer (in patients who cannot use fam-trastuzumab deruxtecan-nxki [Enhertu]).

If approved dato-DXd will be the first TPOP2-targeted therapy for NSCLC and the second for breast cancer. It may directly compete with Trodelvy for the treatment HR-positive/HER2-negative breast cancer in patients who cannot take Enhertu. In clinical trials, dato-DXd demonstrated a favorable and manageable safety profile. While Trodelvy carries a boxed warning for severe or life-threatening neutropenia and severe diarrhea (grade ≥ 3 incidence: neutropenia 49%, diarrhea 11%), these adverse events were reported in a very small number of patients treated with dato-DX in clinical trials.

Dato-DXd is also in phase 3 trials for use in combination with durvalumab (Imfinzi), with or without chemotherapy, in patients with NSCLC, triple-negative breast cancer, or HR-low/HER2-negative breast cancer. Trodelvy is also in phase 3 trials for NSCLC and is currently approved for urothelial carcinoma.

FDA approval timeline

NSCLC - Dec. 20, 2024
Breast Cancer - Jan. 29, 2025

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales*	$49	$425	$798	$1,141	$1,557

_{*Includes sales for NSCLC and breast cancer}

tabelecleucel IV

Manufacturer: Pierre Fabre/Atara

Proposed indications

Monotherapy treatment of Epstein-Barr virus-positive (EBV+) post-transplant lymphoproliferative disease (PTLD) in patients ≥ 2 years of age who have received at least one prior therapy (this includes chemotherapy, unless inappropriate, in solid organ transplant recipients).

Clinical overview

Mechanism of action

Tabelecleucel (tab-cel) is an allogeneic, Epstein-Barr virus (EBV)-specific T-cell immunotherapy that kills EBV infected cells in a targeted manner.

Clinical trials

The global, open-label, phase 3 ALLELE trial (NCT03394365) evaluated tab-cel in 43 patients with EBV+ PTLD following HSCT or solid organ transplant (SOT). Participants had EBV+ PTLD that was refractory to or relapsed after rituximab (after HSCT) or rituximab and chemotherapy (after SOT). The ORR was 51.2% (50% in HSCT cohort, 52% in SOT cohort). The median DOR was 23 months, and the median OS was 18.4 months. At one year, the OS rate was 84.4% among responders compared to 34.8% among non-responders. Tab-cel was generally well tolerated, with no episodes of CRS, immune effector cell-associated neurotoxicity syndrome or transplant rejection related to tab-cel.

Dosage and administration

In the clinical trial, patients received tab-cel administered IV over 5 to 10 minutes at a dose of 2 × 10⁶ cells/kg on days one, eight and 15 in 35-day cycles. Treatment continued until maximal response, unacceptable toxicity, initiation of non-protocol therapy, or failure of tab-cel. The median number of treatment courses received was two cycles in the SOT cohort and three cycles in the HSCT cohort.

Place in therapy

PTLD is an ultra-rare, acute, life-threatening, hematologic malignancy that can occur after SOT or an allogeneic HSCT. It typically appears in the first year after transplantation in patients with weakened T cell activity due to immunosuppressive therapy. The reported incidence of PTLD ranges from 1% to 30% for SOT, depending on organ type, and about 3% for HSCT. While HSCT recipients have a limited time during which they are at risk for PTLD, SOT recipients carry a long-term risk of developing PTLD since they require lifelong immunosuppression. Ebstein-Barr virus is implicated in the majority of PTLD cases. The most significant risk factor for developing PTLD is a transplant involving an EBV-seropositive donor and an EBV-seronegative recipient. Pediatric transplant recipients are more susceptible to developing PTLD than adult recipients, as roughly 95% of all adults over 40 years of age have been exposed to EBV. High-dose post-transplant immunosuppression therapy is also associated with the development of PTLD.

Treatment for PTLD includes a reduction in immunosuppression therapy for early EBV+ PTLD lesions. If a complete response is not achieved, off-label use of rituximab with or without chemotherapy is initiated and results in a response in about 50% to 60% of patients. Among patients who relapse or are refractory to treatment, options are limited, and survival is poor. Non-pharmacologic treatments include surgery to remove localized lesions, and radiation therapy for localized disease or CNS involvement. Adoptive immunotherapy involving EBV-specific cytotoxic T lymphocytes (EBV-CTLs), or donor lymphocyte infusion is also used but may increase the risk for GVHD, and is therefore, reserved for EBV+ PTLD cases that persists despite initial treatment.

If approved, tab-cel will be the first treatment specifically indicated for EBV+ PTLD. It will likely be used as a second-line treatment following rituximab. The therapy is manufactured from T cells collected from healthy EBV+ donors. The cells are enhanced to recognize EBV and expanded to produce thousands of doses from a single donor. The resulting cells are characterized by EBV-specific cytotoxicity, alloreactivity and phenotype to establish a tab-cel product inventory. While tab-cel is an off-the-shelf therapy, it requires individualization based on patient disease criteria including human leukocyte antigen (HLA)-matching. The use of EBV-targeted cells, along with partial HLA matching, reduces the risk of off-target binding and host rejection. In addition, tab-cel’s diverse inventory allows for patients who do not initially respond to tab-cel to be switched to another product within the tab-cel library. 

As an allogeneic T-cell therapy, tab-cel offers potential advantages over autologous CAR T therapies. Unlike CAR T therapy, tab-cel is readily available and administered over a few minutes compared to a 30-minute infusion with autologous CAR T. In addition, tab-cel does not require the patient to undergo apheresis or lymphodepletion and is not associated with CRS or neurotoxicity. Tab-cel may be more accessible as it requires a shorter monitoring time period during and after the dose (1 to 2 hours) in a controlled setting equipped to handle adverse reactions compared to one to two weeks of inpatient monitoring with autologous CAR T therapy.

Notably, ICER released draft evidence reports on tab-cel for EBV+ PTLD that will be reviewed by the New England Comparative Effectiveness Public Advisory Council (CEPAC) on Nov. 14, 2024. The final evidence report is anticipated in December 2024.

FDA approval timeline

Jan. 15, 2025

FDA designations: Breakthrough Therapy, Orphan Drug, Priority Review

Financial forecast (reported in millions)

The financial forecast for tradipitant is not currently available.

revakinagene taroretcel (NT-501) intravitreal implant

Manufacturer: Neurotech

Proposed indications

Macular telangiectasia (MacTel) type 2

Clinical overview

Mechanism of action

Revakinagene taroretcel is an encapsulated cell therapy (ECT) surgically inserted into the ocular vitreous. It has allogeneic retinal pigment epithelial cells genetically modified to produce recombinant ciliary neurotrophic factor (CNTF), which is normally secreted by neurons and Müller cells of the eye. CNTF has demonstrated a neuroprotective effect with the ability to reduce the loss of photoreceptor cells.

Clinical Trials

Two identical international, double-blind, phase 3 trials, NTMT-03-A (NCT03316300; n=115) and NTMT-03-B (NCT03319849, n=113), evaluated revakinagene taroretcel in patients with MacTel type 2 in at least one eye. Enrolled patients had ellipsoid zone (EZ) loss between 0.16 mm² and 2 mm²; EZ area loss indicates diminished vision through loss of photoreceptors. Patients were randomized 1:1 to receive revakinagene taroretcel or sham procedure in one study eye. Both studies reported a statistically significant change in EZ area loss from baseline through 24 months (primary endpoint) with revakinagene taroretcel relative to sham (reduction in EZ area loss: 56.4% in NTMT-03-A [p<0.0001] and 29.2% in NTMT-03-B [p=0.021]). Preserved retinal sensitivity and monocular reading speed at 24 months (secondary endpoints) were also demonstrated with revakinagene taroretcel. Serious ocular TEAEs were reported in ≤ 7% of patients who received revakinagene taroretcel in each trial and were primarily associated with the surgical procedure and were transient in nature. In trial NTMT-03-A, uveitis/iridocyclitis was reported in 9% of patients who received revakinagene taroretcel and 2% who received the sham procedure.

Dosage and administration

In clinical trials, revakinagene taroretcel was inserted intravitreally during a single outpatient surgical procedure.

Place in therapy

MacTel type 2 is a rare, neurodegenerative disorder that affects the macula, a central zone of the retina responsible for detailed vision. MacTel type 2 is characterized by alterations in macular capillaries and photoreceptor atrophy. This may lead to blurred or distorted vision and loss of central vision. MacTel type 2 may also exhibit features similar to wet AMD, such as the development of abnormal blood vessels under the retina (neovascularization). MacTel type 2 is typically diagnosed in middle age (40 to 60 years) and progresses bilaterally over a period of 10 to 20 years. Prevalence is estimated between 0.0045% and 0.1% among those 40 years of age and older. Estimated incidence in the U.S. has been reported as 0.7 per 100,000 persons per year. The exact cause of MacTel type 2 is unknown, but diabetes and hypertension may increase the risk of its development.

In MacTel type 2, Müller cells do not function properly, which leads to degeneration of photoreceptor cells. The extent of vision loss varies, and many patients may not require treatment. However, some patients with vision loss, particularly those with photoreceptor loss in the fovea (central area of the macula), may experience a significant impact on their QOL. There is no FDA-approved treatment for MacTel type 2. Corticosteroid injections have been used to reduce inflammation in the eye and intravitreal injections of anti-vascular endothelial growth factor (anti-VEGF) agents have been used in patients with neovascularization. However, no available treatment, including laser therapy, offers a significant improvement in vision.

If approved, revakinagene taroretcel will be the first treatment indicated for MacTel type 2. It is a first-in-class treatment that uses a new ECT technology to promote continuous, long-term delivery of CNTF in the eye; consistent CNTF levels were observed over a 14.5-year period with revakinagene taroretcel. In clinical trials, revakinagene taroretcel demonstrated the ability to preserve photoreceptors by up to 56% compared to sham. However, like other available treatments, it has not been shown to offer a significant improvement in vision.

FDA approval timeline

Dec. 14, 2024

FDA Designations: Fast Track, Orphan Drug, Priority Review

Financial forecast (reported in millions)

The financial forecast for revakinagene taroretcel is not currently available.

vanzacaftor/tezacaftor/deutivacaftor oral

Manufacturer: Vertex

Proposed indications

Cystic fibrosis (CF) in patients ages ≥ 6 years with at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene

Clinical overview

Mechanism of action

Vanzacaftor/tezacaftor/deutivacaftor (vanza triple) contains three CFTR modulators, including the CFTR correctors vanzacaftor and tezacaftor and the CFTR potentiator deutivacaftor. CFTR correctors facilitate the processing and trafficking of the CFTR protein to increase the amount of CFTR protein at the cell surface. The CFTR potentiator increases the channel open probability (gating) of the CFTR protein at the cell surface to improve the flow of salt and water across the cell membrane.

Clinical trials

The double-blind, active-controlled, 52-week SKYLINE 102 (n=398; NCT05033080) and SKYLINE 103 (n=573; NCT05076149) trials compared the efficacy of once-daily vanza triple to twice-daily doses of elexacaftor/tezacaftor/ivacaftor (Trikafta) in patients ≥ 12 years of age with CF and at least one F508del mutation or a mutation responsive to triple combination CFTR modulators. All patients received Trikafta during a four-week run-in period prior to randomization. In both trials, vanza triple demonstrated non-inferiority to Trikafta based on the primary endpoint o f absolute change from baseline in percent predicted FEV₁ (ppFEV₁) through week 24 (LS mean difference of 0.2 reported in both studies; p<0.0001). In both trials, vanza triple was superior to Trikafta in key secondary endpoints around sweat chloride (SwCl) parameters, including reduction in SwCl levels at week 24 (LS mean difference: SKYLINE 102, -8.4 [p<0.0001]; SKYLINE 103, -2.8 [p=0.0034]). Vanza triple was well tolerated, with a safety profile similar to Trikafta.

The ongoing, single-arm, open-label RIDGELINE (NCT05422222) safety study enrolled 78 patients six to 11 years of age with CF and at least one F508del mutation or a mutation responsive to triple combination CFTR modulator therapy. The safety profile for vanza triple for patients six to 11 years of age was like that reported in patients ≥ 12 years of age. In addition, vanza triple did not result in an improvement in ppFEV₁ (LS mean change, 0.0) from baseline to week 24, but reduced the SwCl levels compared to a baseline level on Trikafta (LS mean change of -8.6 mmol/L).

Dosage and administration

In the clinical trials, vanza triple was administered orally once daily in the morning as a fixed dose combination tablet containing vanzacaftor 20 mg, tezacaftor 100 mg and deutivacaftor 250 mg.

Place in therapy

CF is a serious autosomal recessive multiorgan disorder that affects nearly 40,000 people in the U.S., with approximately 1,000 new cases diagnosed each year. Prevalence is highest in Caucasians with northern European ancestry (1 in 3,200-3,500 population). Most patients with CF are diagnosed by two years of age (median age, six to eight months). In CF, mutations in the CTFR gene cause a decreased quantity and/or function of the CFTR protein. This leads to the accumulation of thick mucus in the lungs, pancreas, pancreas, and other organs. The F508del mutation is the most common mutation associated with CF, present in nearly 90% of CF patients, about 50% of which have two copies of the mutation (homozygous); another 25% to 30% have one copy of the F508del mutation plus another mutation (heterozygous).

The median predicted survival of individuals with CF continues to improve due to advancements in treatment, most notably the development of CFTR-directed therapy. The current median predicted survival is 38 years for those born between 2004–2008, prior to the availability of CFTR modulators in the U.S. However, it has increased to 45 years of age among patients born between 2014–2018 and to 61 years of age in those born between 2019–2023. This coincides with the FDA approval of the first CFTR modulator in 2012 (ivacaftor [Kalydeco]), followed by dual therapy in 2015 and 2018 (lumacaftor/ivacaftor [Orkambi] and tezacaftor/ivacaftor [Symdeko], respectively), and the first triple therapy in 2019 (Trikafta). Moreover, based on age or genotype, 92% of patients with CF were eligible for CFTR modulator therapy in 2023 compared to only 60% in 2018, and 76% of patients prescribed a CFTR modulator received triple therapy with Trikafta. Notably, there has also been a dramatic decrease in the number of lung transplants in individuals with CF since the availability of triple combination therapy.

Fixed-dose vanza triple contains vanzacaftor, a next generation CFTR corrector, tezacaftor, a first-generation CFTR corrector and deutivacaftor, a once-daily CFTR potentiator. In clinical trials, once daily dosing of vanza triple was noninferior in improving lung function (ppFEV₁) and superior in lowering SwCl levels compared to twice-daily Trikafta. Trikafta is indicated to treat CF in patients ≥ 2 years of age who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive. If vanza triple is approved, Vertex expects that most patients on Trikafta, also by Vertex, ≥ 6 years of age may switch to vanza triple due to vanza triple’s improved efficacy in lowering SwCl and desirable once-daily dosing regimen.

FDA approval timeline

Jan. 2, 2025

FDA Designations: Fast Track, Orphan Drug, Priority Review

Financial forecast (reported in millions)

Year	2024	2025	2026	2027	2028
Projected Yearly U.S. Sales	$0	$1,079	$2,193	$3,325	$4,160

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 2028, are displayed. The financials are projected total annual U.S. sales, reported in millions.

Name	Manufacturer	July2024 Pipeline - Total US sales for 2028 (Dollars in millions)
aficamten	Cardiovascular	$1,152
botaretigene sparoparvovec	Ophthalmology / Gene therapy	$121
etripamil (Cardamyst)	Cardiovascular	$134
inavolisib	Oncology	$408
marstacimab	Hematology	$197
mirdametinib	Oncology	$332
nipocalimab	Immunology	$537
patritumab deruxtecan	Oncology	$438
prademagene zamikeracel (EB-101)	Dermatology / Gene therapy	$216
remestemcel-L (Ryoncil)	Immunology / Cellular	$263
revumenib	Oncology	$370
RGX-121	Metabolic / Gene therapy	$71
sebetralstat	Immunology	$331
vimseltinib	Oncology	$30
vusolimogene oderparepvec	Oncology / Gene therapy	$230
zenocutuzumab	Oncology	$82

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 & cellular therapies

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
eladocagene exuparvovec (Upstaza)	PTC	Aromatic L-amino acid decarboxylase (AADC) deficiency	Intraputaminal	BLA; seeking Accelerated Approval, Orphan Drug, Priority Review, RPD	11/13/2024
obecabtagene autoleucel	Autolus	ALL (R/R, B-cell, adults)	IV	BLA; Orphan Drug, RMAT	11/16/2024
revakinagene taroretcel (NT-501)	Neurotech	Macular telangiectasia type 2	Intravitreal implant	BLA; Fast Track, Orphan Drug, Priority Review	12/17/2024
remestemcel-L	Mesoblast	Acute GVHD (steroid-refractory, children)	IV	BLA; Fast Track, Orphan Drug	01/07/2025
tabelecleucel	Pierre Fabre/Atara	Epstein-Barr virus-associated post-transplant lymphoproliferative disease	IV	BLA; Breakthrough Therapy, Orphan Drug, Priority Review	01/15/2025

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	Candel	Prostate cancer	Intratumorally	BLA; Fast Track	TBD
botaretigene sparoparvovec	Janssen	Retinitis pigmentosa	Subretinal	BLA; Fast Track, Orphan Drug	TBD
deramiocel	Nippon Shinyaku	DMD cardiomyopathy	IV	BLA; Orphan Drug, RMAT, RPD	TBD
donaperminogene seltoplasmid	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
prademagene zamikeracel	Abeona	Epidermolysis bullosa (recessive dystrophic)	Topical	BLA; Breakthrough Therapy, Orphan Drug, RMAT, RPD	TBD
RGX-121	Regenxbio	Mucopolysaccharidosis II (Hunter syndrome)	Intrathecal	BLA; may seek Accelerated Approval, Fast Track, Orphan Drug, RMAT, RPD	TBD
rilparencel (React)	Prokidney	CKD	Hepatic injection	BLA; RMAT	TBD
vusolimogene oderparepvec	Replimune	Melanoma (anti-PD1 failed)	Intratumorallytd>	BLA	TBD

None

Biosimilars

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
denosumab (biosimilar to Amgen's Prolia/Xgeva	Celltrion	Osteoporosis/osteopenia	SC	BLA	12/01/2024
ustekinumab (biosimilar to Janssen's Stelara)	Biocon/Janssen	PSO; PsA; CD; UC	IV, SC	BLA	12/27/2024
trastuzumab (biosimilar to Genentech's Herceptin)	Tanvex	Breast cancer (HER2+); Gastric cancer (HER2+)	IV	BLA	01/06/2025
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	01/28/2025
ustekinumab (biosimilar to Janssen's Stelara)	Bio-Thera Solutions	PSO; PsA; CD; UC	IV, SC	BLA	May-Jun 2025
denosumab (biosimilar to Amgen's Prolia/Xgeva)	Teva	Osteoporosis/osteopenia	SC	BLA	Jul-Dec 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 and Lee/Sandoz	T1DM; T2DM	SC	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
treosulfan	Medexus	Allogeneic HSCT (preparation, in combination with fludarabine)	IV	NDA; Orphan Drug	Oct 2024
garadacimab	CSL	HAE	SC	BLA; Fast Track, Orphan Drug	Oct-Nov 2024
inavolisib	Genentech	Breast cancer (HR+/HER2-, 1st-line, in combination with palbociclib and fulvestrant)	Oral	NDA; Breakthrough Therapy, Priority Review	11/27/2024
govorestat	Applied Therapeutics	Classic galactosemia	Oral	NDA; Fast Track, Orphan Drug, Priority Review, RPD	11/28/2024
acoramidis	Bridgebio/AstraZeneca	Transthyretin amyloid cardiomyopathy (ATTR-CM)	Oral	NDA; Orphan Drug	11/29/2024
zanidatamab	Jazz	Biliary tract cancer (unresectable, locally advanced or metastatic, HER2+, previously-treated)	IV	BLA; seeking Accelerated Approval, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review	11/29/2024
concizumab	Novo Nordiska	Hemophilia A and B (with inhibitors)	SC	BLA; Breakthrough Therapy, Orphan Drug	Dec 2024
olezarsen	Akcea/Ionis	Familial chylomicronemia syndrome	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review	12/19/2024
datopotamab deruxtecan	Daiichi Sankyo/AstraZeneca	NSCLC (locally advanced or metastatic, nonsquamous, ≥ 2nd-line)	IV	BLA	12/20/2024
glepaglutide	Zealand	Short bowel syndrome (with intestinal failure)	SC	NDA; Orphan Drug	12/20/2024
revumenib	Syndax/Abbvie	Acute leukemia (R/R KMT2A-rearranged)	Oral	NDA; Breakthrough Therapy, Orphan Drug, Priority Review, RTOR	12/26/2024
diazoxide choline	Soleno	Prader-Willi syndrome (ages ≥ 4 years with hyperphagia)	Oral	505(b)(2) NDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review	12/27/2024
nivolumab/hyaluronidase (Opdivo SC)	Bristol Myers Squibb	Bladder cancer; Melanoma; RCC; Solid Tumors	SC	BLA	12/27/2024
cosibelimab	Checkpoint	Cutaneous squamous cell carcinoma (metastatic or locally advanced, eligible for curative surgery or radiation)	IV	BLA	12/28/2024
ensartinib	Xcovery	NSCLC (metastatic, ALK+)	Oral	NDA	12/28/2024
crinecerfont	Neurocrine	Classic congenital adrenal hyperplasia	Oral	NDA; Breakthrough Therapy, Orphan Drug, Priority Review	12/29/2024
bentracimab	SERB	Ticagrelor (Brilinta) reversal	IV	BLA; Breakthrough Therapy, Priority Review	Jan-Mar 2025
mitomycin	Urogen	Bladder cancer (low-grade intermediate-risk non-muscle invasive)	Intravesicular	505(b)(2) NDA	Jan-Jun 2025
vanzacaftor/tezacaftor/deutivacaftor	Vertex	CF (ages ≥ 6 years, ≥ 1 F506del mutation or other responsive CFTR gene mutation)	Oral	NDA; Fast Track, Orphan Drug, Priority Review	01/02/2025
zenocutuzumab	Merus	NSCLC (NRG1+); Pancreatic cancer (NRG1+)	IV	BLA; Breakthrough Therapy, Orphan Drug, Priority Review	01/06/2025
datopotamab deruxtecan	Daiichi Sankyo/AstraZeneca	Breast cancer (unresectable or metastatic, HR+/HER2- [IHC 0, IHC 1+ or IHC 2+/ISH-])	IV	BLA	01/29/2025
elamipretide	Stealth	Barth syndrome	SC	NDA; Fast Track, Orphan Drug, Priority Review, RPD	01/29/2025
apomorphine	Supernus	Parkinson's disease (continuous treatment of motor fluctuations)	SC infusion	NDA	01/31/2025
amivantamab/hyaluronidase (Rybrevant SC)	Janssen	NSCLC	SC infusion	BLA; Priority Review	02/17/2025
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
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	03/20/2025
fitusiran	Sanofi	Hemophilia A and B (with or without inhibitors	SC	NDA; Fast Track, Orphan Drug	03/28/2025
atrasentan	Novartis/Abbvie	IgA nephropathy (Berger's disease)	Oral	NDA; seeking Accelerated Approval	May-Jun 2025
condoliase	Ferring	Radicular leg pain associated with lumbar disc herniation	Intrathecal	BLA	05/14/2025
concizumab	Novo Nordisk	Hemophilia A and B (without inhibitors)	SC	BLA; Breakthrough Therapy, Orphan Drug	05/31/2025
sebetralstat	Kalvista	HAE (on-demand, ages ≥ 12 years)	Oral	NDA; Fast Track, Orphan Drug	06/17/2025
chenodiol	Mirum	Cerebrotendinous xanthomatosis	Oral	NDA; Orphan Drug	06/28/2025
delgocitinib	Leo	Chronic hand eczema (corticosteroid failure/inappropriate)	Topical	NDA; Fast Track	Jul-Sep 2025
sepiapterin	PTC	Phenylketonuria (PKU)	Oral	NDA; Orphan Drug	07/30/2025
ataluren	PTC	DMD (nonsense mutation)	Oral	NDA; Fast Track, Orphan Drug	07/31/2025
nipocalimab	Janssen	Myasthenia gravis	IV	BLA; Fast Track, Orphan Drug	08/29/2025
paltusotine	Crinetics	Acromegaly	Oral	NDA; Orphan Drug	09/26/2025
telisotuzumab vedotin	Abbvie	NSCLC (locally advanced/metastatic, EGFR wild type, nonsquamous, c-Met protein overexpression)	IV	BLA; seeking Accelerated Approval, Breakthrough Therapy	09/26/2025
sodium chlorite	Neuvivo	ALS	IV	NDA; Orphan Drug	10/07/2025

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
fam-trastuzumab deruxtecan-nxki (Enhertu)	Daiichi Sankyo/AstraZeneca	Breast cancer (HER2+, 3rd-line)	IV	sBLA; Breakthrough Therapy, Fast Track	Oct-Dec 2024
olaparib (Lynparza)	AstraZeneca	Endometrial cancer (1st-line, in combination with durvalumab)	Oral	sNDA	Oct-Dec 2024
tislelizumab-jsgr (Tevimbra)	Beigene	Esophageal squamous cell carcinoma (unresectable, recurrent, locally advanced, or metastatic, 1st-line)	IV	sBLA; Orphan Drug	Oct-Dec 2024
bimekizumab-bkzx (Bimzelx)	UCB	Hidradenitis suppurativa (moderate to severe)	SC	sBLA	Nov-Dec 2024
avacincaptad pegol (Izervay)	Astellas	Dry AMD-related geographic atrophy	Intravitreal	sNDA; Breakthrough Therapy, Fast Track	11/19/2024
durvalumab (Imfinzi)	AstraZeneca	SCLC (limited stage, progressed following platinum-based concurrent chemoradiotherapy)	IV	sBLA; Breakthrough Therapy, Orphan Drug, Priority Review	Dec 2024
tislelizumab-jsgr (Tevimbra)	Beigene	Gastric or gastroesophageal junction adenocarcinoma (locally advanced unresectable or metastatic, in combination with fluoropyrimidine- and platinum-containing chemotherapy)	IV	sBLA	Dec 2024
nemolizumab-ilto (Nemluvio)	Galderma	Atopic dermatitis (moderate-severe, adults and adolescents)	SC	sBLA	12/14/2024
setmelanotide (Imcivree)	Rhythm	Bardet-Biedl or pro-opiomelanocortin related obesity (ages ≥ 2 years)	SC	sNDA; Breakthrough Therapy, Orphan Drug, Priority Review, RPD	12/26/2024
acalabrutinib (Calquence)	AstraZeneca	Mantle cell lymphoma (1st-line)	Oral	sNDA; Breakthrough Therapy, Priority Review	Jan-Mar 2025
lecanemab-irmb (Leqembi)	Eisai	Alzheimer's disease (mild, monthly maintenance dosing)	IV	sBLA; Breakthrough Therapy, Fast Track	01/25/2025
asciminib (Scemblix)	Novartis	CML (Ph+, chronic phase, 1st-line)	Oral	sNDA; Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review	01/29/2025
mirikizumab-mrkz (Omvoh)	Eli Lilly	CD	IV, SC	sBLA	01/31/2025
elexacaftor/tezacaftor/ivacaftor (Trikafta)	Vertex	CF (rare responsive mutations)	Oral	sNDA; Breakthrough Therapy, Fast Track, Orphan Drug	Feb-Apr 2025
fam-trastuzumab deruxtecan-nxki (Enhertu)	Daiichi Sankyo/AstraZeneca	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
brentuximab vedotin (Adcetris)	Pfizer	Large B-cell lymphoma (R/R, in combination with lenalidomide & rituximab)	IV	sBLA; Orphan Drug	Mar 2025
furosemide (Furoscix)	scPharmaceuticals	CKD-related edema	SC	sNDA	03/06/2025
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
cobicistat/darunavir (Prezcobix)	Janssen	HIV-1 infection (ages ≥ 6 years, weighing ≥ 25 kg)	Oral	sNDA	04/04/2025
vutrisiran (Amvuttra)	Alnylam	Transthyretin amyloid cardiomyopathy (ATTR-CM)	SC	sBLA; Orphan Drug, Priority Review	04/09/2025
guselkumab (Tremfya)	Janssen/Novartis	CD (moderate to severe)	IV, SC	sBLA	04/18/2025
ipilimumab (Yervoy)	Bristol Myers Squibb	HCC (unresectable, 1st-line, in combination with nivolumab)	IV	sBLA	04/21/2025
nivolumab (Opdivo)	Bristol Myers Squibb	HCC (unresectable, 1st-line, in combination with ipilimumab)	IV	sBLA	04/21/2025
ranibizumab port delivery system (Susvimo)	Genentech	DME; Diabetic retinopathy	Intravitreal implant	sBLA	May-Jun 2025
esketamine (Spravato)	Janssen	MDD (treatment-resistant, monotherapy)	Intranasal	sNDA; Breakthrough Therapy, Fast Track	05/22/2025
upadacitinib (Rinvoq	Abbvie	Giant cell arteritis	Oral	sNDA	07/11/2025
darolutamide (Nubeqa)	Bayer AG	Prostate cancer (metastatic, hormone-sensitive, in combination with androgen deprivation therapy [ADT])	Oral	sNDA; Fast Track	07/25/2025
daratumumab/hyaluronidase-fihj (Darzalex Faspro)	Janssen	Multiple myeloma (newly diagnosed, in combination with bortezomib, lenalidomide and dexamethasone, autologous SCT deferred or ineligible.	SC	sBLA	07/30/2025
lonapegsomatropin-tcgd (Skytrofa)	Ascendis	Growth hormone deficiency (adults)	SC	sBLA	07/30/2025

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
aficamten	Cytokinetics	Hypertrophic cardiomyopathy	Oral	NDA; Breakthrough Therapy, Orphan Drug	TBD
amlitelimab	Sanofi	Atopic dermatitis	SC	BLA	TBD
antiBKV	Memo	BK polyomavirus (BKV) infection	IV	BLA; Fast Track	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	COPD	IV	BLA; Fast Track	TBD
avutometinib	Verastem	Ovarian cancer (recurrent KRAS-mutant, low-grade, serous, in combination with defactinib, ≥ 1 prior systemic therapy)	Oral	NDA; may seek Accelerated Approval, Breakthrough Therapy, Orphan Drug	TBD
azemiglitazone	Cirius	MASH	Oral	NDA	TBD
azetukalner	Xenon	Focal-onset seizures	Oral	NDA	TBD
belapectin	Galectin	MASH	IV	NDA; Fast Track	TBD
bevacizumab-vikg	Outlook	Wet AMD	Intravitreal	BLA	TBD
cabozantinib	Exelixis	Prostate cancer (metastatic, castration-resistant)	Oral	NDA	TBD
cetuximab sarotalocan	Rakuten	SCCHN	IV	BLA; Fast Track	TBD
clesrovimab	Merck	RSV prevention (infants)	IM	BLA	TBD
cobolimab	GlaxoSmithKline	NSCLC	IV	BLA	TBD
defactinib	Verastem/Pfizer	Ovarian cancer (KRAS-mutant, low-grade, serous, in combination with avutometinib, ≥ 1 prior systemic therapy)	Oral	NDA; may seek Accelerated Approval, Breakthrough Therapy, 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	Neuroendocrine tumors	IV	BLA; Orphan Drug	TBD
donidalorsen	Ionis	HAE	SC	NDA; Orphan Drug	TBD
fazirsiran	Arrowhead	Alpha-1 antitrypsin deficiency	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
fenebrutinib	Genentech	MS	Oral	NDA	TBD
fianlimab	Regeneron	Melanoma	IV	BLA; Fast Track	TBD
ganaxolone (Ztalmy IV)	Marinus	Tuberous sclerosis complex	IV	NDA; Orphan Drug	TBD
gemcitabine-releasing intravesical system (TAR-200)	TARIS/Janssen	Bladder cancer (non-muscle invasive)	Intravesicular	NDA; Breakthrough Therapy, Fast Track	TBD
giredestrant	Genentech	Breast cancer (HR+/HER2-)	Oral	NDA; Fast Track	TBD
gold nanocrystal	Clene	ALS	Oral	NDA; Orphan Drug	TBD
govorestat	Applied	Sorbitol dehydrogenase (SORD) deficiency	Oral	NDA; Orphan Drug	TBD
hydroxypropyl beta cyclodextrin	Cyclo	Niemann-Pick disease type C	IV	NDA; Fast Track, Orphan Drug, RPD	TBD
ianalumab	Novartis	Autoimmune hepatitis; Sjogren's syndrome; ITP	SC	BLA; Fast Track	TBD
imlifidase	Sarepta	Kidney transplant rejection	IV	BLA; Fast Track, Orphan Drug	TBD
inaxaplin	Vertex	Focal segmental glomerulosclerosis (APOL1-mediated)	Oral	NDA; may seek Accelerated Approval, Breakthrough Therapy	TBD
itepekimab	Regeneron	COPD	SC	BLA; Fast Track	TBD
JDQ-443	Novartis	NSCLC	Oral	NDA	TBD
ketamine (NRX100)	Hope/NRx	MDD (suicidal)	IV	505(b)(2) NDA; Fast Track	TBD
lanifibranor	Inventiva	MASH	Oral	NDA; Breakthrough Therapy, Fast Track	TBD
latozinemab	Alector/GlaxoSmithKline	Frontotemporal dementia	IV	BLA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
lerodalcibep	LIB	Dyslipidemia; HeFH; HoFH	SC	BLA	TBD
ligelizumab	Novartis	Food allergies	SC	BLA	TBD
linerixibat	GlaxoSmithKline	Primary biliary cholangitis	Oral	NDA; Orphan Drug	TBD
molgramostim	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	IV	BLA; Fast Track	TBD
nerandomilast	Boehringer Ingelheim	Idiopathic pulmonary fibrosis	Oral	NDA; Breakthrough Therapy, Orphan Drug	TBD
obefazimod	Abivax	UC (moderately-severely active)	Oral	NDA	TBD
pabinafusp alfa	JCR	Mucopolysaccharidosis II (Hunter syndrome)	IV	BLA; Orphan Drug	TBD
palazestrant	Olema	Breast cancer (HR+/HER2-)	Oral	NDA; Fast Track	TBD
patritumab deruxtecan	Merck	NSCLC ( locally advanced or metastatic, EGFR-mutated, ≥ 2 prior systemic therapies)	IV	BLA; may seek Accelerated Approval, Breakthrough Therapy	TBD
pegadricase	Swedish Orphan Biovitrum	Gout	IV	BLA	TBD
pegargiminase	Polaris	Mesothelioma	IM	BLA; Fast Track, Orphan Drug	TBD
pegzilarginase	Immedica	Arginase 1 deficiency (ARG1-D)	IV	BLA; Breakthrough Therapy, Fast Track, Orphan Drug, RPD	TBD
pelabresib	Novartis	Myelofibrosis	Oral	NDA; Fast Track, Orphan Drug	TBD
pelacarsen	Novartis	CVD (elevated lipoprotein[a])	SC	NDA; Fast Track	TBD
plozasiran	Arrowhead	Familial chylomicronemia syndrome	SC	NDA; Breakthrough Therapy, Fast Track, Orphan Drug	TBD
QRX-003	Quoin	Netherton syndrome	Topical	BLA	TBD
REGN-5713-5714-5715	Regeneron	Birch allergy	SC	BLA	TBD
relacorilant	Corcept	Cushing's syndrome; Ovarian cancer	Oral	NDA; Orphan Drug	TBD
remibrutinib	Novartis	MS; Urticaria	Oral	NDA	TBD
resiniferatoxin	Grunenthal	Osteoarthritis pain (knee)	Intra-articular	NDA; Breakthrough Therapy	TBD
rexlemestrocel-L (Revascor)	Mesoblast Limited	Ischemic HFrEF (adults, end-stage, with left ventricular assist device (LVAD) implantation)	Intramyocardial injection	BLA; may seek Accelerated Approval, Orphan Drug, RMAT	TBD
rilzabrutinib	Sanofi	ITP	Oral	NDA; Fast Track, Orphan Drug	TBD
rusfertide	Takeda	Polycythemia vera	SC	NDA; Fast Track, Orphan Drug	TBD
savolitinib	AstraZeneca	NSCLC	Oral	NDA; Fast Track	TBD
secukinumab	Novartis	Giant cell arteritis	SC	BLA	TBD
sefaxersen	Ionis/Roche	IgA nephropathy (Berger’s disease)	SC	NDA	TBD
serplulimab	Henlius	SCLC	IV	BLA; Orphan Drug	TBD
soticlestat	Takeda	Dravet syndrome; Lennox-Gastaut syndrome	Oral	NDA; Orphan Drug	TBD
sozinibercept	Opthea	Wet AMD	Intravitreal	BLA; Fast Track	TBD
sunvozertinib	Dizal (Jiangsu)	NSCLC	Oral	NDA; Breakthrough Therapy	TBD
tamibarotene	Syros	Myelodysplastic syndrome (newly diagnosed, higher-risk, RARA gene overexpression)	Oral	NDA; Fast Track, Orphan Drug	TBD
tiragolumab	Genentech	Esophageal cancer; NSCLC (1st-line)	IV	BLA; Breakthrough Therapy, Orphan Drug	TBD
tiratricol	Rare Thyroid Therapeutics	Monocarboxylate transporter 8 (MCT8) deficiency	Oral	NDA; Fast Track, Orphan Drug, RPD	TBD
tividenofusp alfa	Denali	Mucopolysaccharidosis II (Hunter syndrome)	IV	BLA; Fast Track, Orphan Drug, RPD	TBD
tolebrutinib	Sanofi	MS	Oral	NDA	TBD
ulotaront	Sumitomo	Schizophrenia	Oral	NDA; Breakthrough Therapy	TBD
valiltramiprosate	Alzheon	Alzheimer's disease (early, APOE4 carriers)	Oral	NDA; Fast Track	TBD
vatiquinone	PTC	Friedreich's ataxia	Oral	NDA; Fast Track, Orphan Drug	TBD
veligrotug	Viridian	Thyroid eye disease (TED)	IV	BLA	TBD
venglustat	Sanofi	Fabry's disease; Gaucher's disease	Oral	NDA; Fast Track, Orphan Drug	TBD
zanidatamab	Jazz	Gastric cancer	IV	BLA; Fast Track, Orphan Drug	TBD
zasocitinib	Takeda	PSO	Oral	NDA	TBD

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
aminolevulinic acid (Ameluz)	Biofrontera	Superficial basal cell carcinoma (sBCC)	Topical	sNDA	TBD
atezolizumab (Tecentriq)	Genentech	Prostate cancer; TNBC	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
dexmedetomidine (Igalmi)	Bioxcel	Alzheimer’s disease-related neuropsychiatric symptoms	Oral transmucosal	sNDA; Breakthrough Therapy	TBD
dupilumab (Dupixent)	Sanofi	Bullous pemphigoid; Urticaria	SC	sBLA; Orphan Drug	TBD
eplontersen (Wainua)	Ionis	Transthyretin amyloid cardiomyopathy (ATTR-CM)	SC	sNDA; Fast Track, Orphan Drug	TBD
iptacopan (Fabhalta)	Novartis	C3 glomerulopathy (C3G); Hemolytic uremic syndrome	Oral	sNDA; Breakthrough Therapy, Orphan Drug, RPD	TBD
lecanemab-irmb (Leqembi)	Eisai	Alzheimer's disease (mild, weekly maintenance dosing)	Oral	sNDA; Breakthrough Therapy, Orphan Drug, RPD	TBD
mepolizumab (Nucala)	GlaxoSmithKline	COPD	SC	sBLA	TBD
mitapivat (Pyrukynd)	Agios	SCD; Thalassemia	Oral	sNDA; Orphan Drug	TBD
mosunetuzumab-axgb (Lunsumio)	Genentech	DLBCL (2nd-line, in combination with polatuzumab vedotin)	SC	sBLA	TBD
obinutuzumab (Gazyva)	Genentech	Lupus nephritis; Membranous nephropathy; SLE	IV	sBLA; Breakthrough Therapy	TBD
pegcetacoplan (Empaveli)	Apellis	C3 glomerulopathy (C3G)	SC	sNDA; Orphan Drug	TBD
pitolisant (Wakix)	Harmony	Idiopathic hypersomnia	Oral	sNDA; Orphan Drug	TBD
retifanlimab-dlwr (Zynyz)	Incyte	Anal cancer	IV	sBLA; Fast Track, Orphan Drug	TBD
secukinumab (Cosentyx)	Novartis	Giant cell arteritis; Lupus nephritis	SC	sBLA	TBD
tafasitamab-cxix (Monjuvi)	Incyte	Follicular lymphoma; Marginal zone lymphoma	IV	sBLA; Orphan Drug	TBD
venetoclax (Venclexta)	Abbvie/Genenetech	Myelodysplastic syndrome	Oral	sNDA; Breakthrough Therapy, Orphan Drug	TBD

Traditional

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
minocycline HCl 40 mg	Journey	Rosacea	Oral	505(b)(2) NDA	11/04/2024
vicagrel	Jiangsu Vcare	Thrombotic cardiovascular and cerebrovascular diseases	Oral	NDA	12/28/2024
lamotrigine oral suspension	OWP	Bipolar disorder; Seizure disorder	Oral	505(b)(2) NDA	01/03/2025
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	NDA	01/31/2025
chikungunya vaccine	Bavarian	Chikungunya virus immunization (ages ≥ 12 years)	IM	BLA; Breakthrough Therapy, Fast Track, Priority Review	02/14/2025
meningococcal vaccine (GSK3536819A)	GlaxoSmithKline	Meningococcal immunization	IM	BLA	02/14/2025
hydrocortisone oral solution (ET-400)	Eton	Adrenocortical insufficiency	Oral	NDA	02/28/2025
gepotidacin	GlaxoSmithKline	UTI (uncomplicated, adult and adolescent females)	Oral	NDA; Priority Review, QIDP	03/26/2025
etripamil	Milestone	Paroxysmal supraventricular tachycardia	Intranasal	NDA	03/26/2025
reproxalap	Aldeyra	DED	Topical	NDA	Apr 2025
AR-15512	Aerie	DED	Ophthalmic	NDA	05/30/2025
oxylanthanum carbonate	Unicycive	Hyperphosphatemia (in patients with CKD on dialysis)	Oral	505(b)(2) NDA	07/03/2025
elinzanetant	Bayer AG	Menopause-associated vasomotor symptoms	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

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
vibegron (Gemtesa)	Sumitovant	Overactive bladder (in men receiving BPH pharmacotherapy)	Oral	sNDA	Oct-Dec 2024
tapinarof (Vtama)	Dermavant	Atopic dermatitis (ages ≥ 2 years)	Topical	sNDA	12/13/2024
tirzepatide (Zepbound)	Eli Lilly	Sleep apnea (in patients with obesity)	SC	sNDA; Fast Track, possible Priority Review	12/15/2024
sotagliflozin (Inpefa)	Lexicon	T1DM (with CKD)	Oral	sNDA	12/20/2024
semaglutide (Ozempic)	Novo Nordisk	Diabetic nephropathy	SC	sNDA	Jan 2025
smallpox and monkeypox vaccine (Jynneos) - freeze-dried formulation	Bavarian	Mpox immunization (adults); Smallpox immunization (adults)	SC	sBLA	Jan-Mar 2025
brexpiprazole (Rexulti)	Otsuka	PTSD (in combination with sertraline)	Oral	sNDA	02/08/2025
roflumilast (Zoryve)	Arcutis	PSO (scalp and body, ages ≥ 12 years)	Topical	sNDA	05/22/2025
epinephrine (Neffy)	ARS	Anaphylaxis (children weighing 15-30 kg)	Intranasal	sNDA	07/09/2025

Name	Manufacturer	Clinical Use	Dosage Form	Development Status	FDA Decision
aroxybutynin/atomoxetine	Apnimed	Sleep apnea	Oral	NDA; Fast Track	TBD
atropine (microdose)	Eyenovia	Pediatric progressive myopia	Ophthalmic	505(b)(2) NDA	TBD
baclofen/naltrexone/sorbitol	Pharnext	Charcot-Marie-Tooth disease	Oral	NDA; Fast Track, Orphan Drug	TBD
blarcamesine	Anavex	Alzheimer’s disease	Oral	NDA	TBD
brensocatib	Insmed	Bronchiectasis	Oral	NDA; Breakthrough Therapy	TBD
brilaroxazine	Reviva	Schizophrenia	Oral	NDA	TBD
cadisegliatin	vTv	T1DM	Oral	NDA; Breakthrough Therapy	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 Life Sciences	Smoking cessation	Oral	NDA	TBD
d-cycloserine/lurasidone	Alvogen	Bipolar disorder (suicidal)	Oral	505(b)(2) NDA; Breakthrough Therapy, Fast Track	TBD
ensitrelvir	Shionogi	COVID-19 treatment	Oral	BLA; Fast Track	TBD
epinephrine	Aquestive	Anaphylaxis	SL	505(b)(2) NDA; Fast Track	TBD
esreboxetine	Axsome/Pfizer	Fibromyalgia	Oral	NDA	TBD
gepotidacin	GlaxoSmithKline	Urogenital gonorrhea	Oral	NDA; QIDP	TBD
hydromethylthionine mesylate	Taurx	Alzheimer's disease (mild-moderate)	Oral	NDA	TBD
Lyme disease vaccine	Valneva/Pfizer	Lyme disease vaccination	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/overweight; T2DM	Oral	NDA	TBD
paromomycin	Appili	Leishmaniasis	Topical	NDA; Orphan Drug	TBD
PL-9643	Palatin	DED	Ophthalmic	NDA; Orphan Drug	TBD
purified vero rabies vaccine (SP0087)	Sanofi	Rabies (post-exposure treatment)	N/A	BLA	TBD
quadrivalent influenza mRNA vaccine (mRNA-1010)	Moderna	Seasonal influenza vaccination	N/A	BLA	TBD
retatrutide	Eli Lilly	Obesity/overweight; T2DM	SC	NDA	TBD
RSV live attenuated vaccine	Sanofi	RSV immunization	Intranasal	BLA	TBD
semaglutide + insulin icodec	Novo Nordisk	T2DM	SC	NDA	TBD
survodutide	Boehringer Ingelheim	Obesity/overweight; T2DM; MASH	SC	NDA	TBD
tebipenem pivoxil	GlaxoSmithKline	UTI (complicated)	Oral	NDA; Fast Track, QIDP	TBD
tradipitant	Vanda/Eli Lilly	Emesis (motion sickness)	Oral	NDA	TBD
tramiprosate	Alzheon	Alzheimer's disease	Oral	NDA; Fast Track	TBD
ulixacaltamide	Praxis	Essential tremor	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	Neuropsychiatric symptoms in Alzheimer’s disease	Oral	sNDA; Breakthrough Therapy, Fast Track	TBD
finerenone (Kerendia)	Bayer AGt	Chronic HFpEF	Oral	sNDA; Breakthrough Therapy, Fast Track	TBD

Name	Manufacturer	Clinical Use	Dosage Form	Development Status
dasatinib (Dasynoc)	Xspray	CML	Oral	CRL
dasiglucagon (Zegalogue)	Novo Nordisk	Congenital hyperinsulinism	SC	CRL
linvoseltamab	Regeneron	Multiple myeloma (4th-line)	IV	CRL
midomafetamine	Lykos	PTSD	SC	CRL
octreotide (Oclaiz)	Camurus	Acromegaly	SC	CRL
tradipitant	Vanda	Gastroparesis	Oral	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

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

APOL1 Apolipoprotein L1

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

BCL-1

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

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

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 Hazard Ratio

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

LS Least Square

LVEF Left Ventricular Ejection Fraction

mAb Monoclonal Antibody

MACE Major Adverse Cardiovascular Events

MADRS Montgomery – Åsberg Depression Rating Scale

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

MI Myocardial Infarction

mITT modified Intent-To-Treat

MRI Magnetic Resonance Imaging

MRSA Methicillin-Resistant Staphylococcus Aureus

MS Multiple Sclerosis

MSI-H Microsatellite Instability-High

mTOR mechanistic Target of Rapamycin

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-Hodgkin Lymphoma

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

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

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

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