Trending Topics & Drug Approvals: September 2025 - Prime Therapeutics
Trending Topics & Drug Approvals: September 2025
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Acetaminophen (Tylenol) update
The United States (U.S.) Food and Drug Administration (FDA) announced the initiation of a safety label change for products containing acetaminophen (Tylenol, other brand names) based on evidence suggesting an association between use during pregnancy and an increased risk for neurological conditions (e.g., autism, attention-deficit/ hyperactivity disorder [ADHD]). The Agency cites large-scale cohort studies (e.g., Nurses’ Health Study II, Boston Birth Cohort) demonstrating this association. Per the FDA, some data suggest chronic use of acetaminophen throughout pregnancy poses the greatest risk. Notably, a causal relationship between acetaminophen and these neurological conditions has not been established and other medical literature refutes the association. The FDA has published a Notice to Physicians recommending health care professionals (HCPs) minimize the use of acetaminophen during pregnancy for routine low-grade fevers. However, the Agency also notes that acetaminophen is the safest over-the-counter (OTC) pain reliever/fever reducer to use in pregnancy as other medications, such as aspirin and ibuprofen, have documented adverse effects on a developing fetus.In response to the FDA, the American College of Obstetricians and Gynecologists (ACOG) has issued a statement affirming the safety and benefits of acetaminophen during pregnancy. ACOG states the FDA/Department of Health and Human Services (HHS) announcements are not supported by the full body of scientific evidence and simplifies the multifactorial, complex causes of neurological problems in children. Furthermore, ACOG states not a single reputable study has succinctly concluded use of acetaminophen in any trimester of pregnancy results in neurodevelopmental disorders in the offspring and the two highest-quality studies on this subject did not find a significant association between use of acetaminophen during pregnancy and the risk of autism, ADHD or intellectual disability in the children. The American Academy of Pediatrics (AAP) also states that research does not show a causal link between acetaminophen use in children and autism and that a single, root cause of autism has not been identified. The AAP has also issued a separate statement refuting the claim that vaccines are linked to autism.
Leucovorin (Wellcovorin) update
The FDA has announced steps towards the approval of leucovorin calcium (Wellcovorin) for patients with cerebral folate deficiency (CFD). Patients with CFD can exhibit seizures, difficulty with movement and coordination as well as developmental delays with autistic features (e.g., social communication challenges, sensory processing issues, repetitious behaviors). The Agency is collaborating with the manufacturer of Wellcovorin (GSK) to develop a process for documentation of the scientific evidence required to demonstrate safety and efficacy of Wellcovorin for pediatric and adult patients with CFD. HHS has also announced the National Institutes of Health (NIH) will launch confirmatory trials as well as new research, including safety studies, on the use of leucovorin. HHS notes that leucovorin is not a cure for autism spectrum disorder (ASD) and may only improve speech-related deficits for a subset of children with ASD. Other non-pharmacological treatments (e.g., behavioral therapy) are also recommended for these children. The NIH has also announced recipients of the Autism Data Science Initiative (ADSI), providing more than $50 million for 13 projects addressing autism.ACIP meeting update
Five new members were appointed to the Center for Disease Control and Prevention’s (CDC) Advisory Committee on Immunization Practices (ACIP) prior to the committee convening in mid-September. ACIP met and discussed measles, mumps, rubella, varicella (MMRV) vaccine, hepatitis B vaccines and coronavirus disease 2019 (COVID-19) vaccines.-
ACIP voted that for children under four years of age, immunization for varicella should occur through a standalone vaccination rather than with the combination of a MMRV vaccine. This decision was based on approximately five more febrile seizures per 10,000 doses in children who received the combination MMRV vaccine compared with receiving separate immunizations for varicella and measles, mumps and rubella (MMR). The standalone varicella vaccine is administered at the same time as the MMR vaccine. Previously, parents/caregivers could choose to administer the MMRV instead of separate MMR and varicella vaccines, if preferred.
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ACIP recommended universal testing of hepatitis B in all pregnant women. The advisory tabled the vote for changing the hepatitis B immunization schedule.
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ACIP also recommended COVID-19 vaccination be determined by individual decision-making for all individuals at least six months of age and older; the HHS states that this immunization approach allows for immunization coverage through all payment mechanisms (e.g., Vaccines for Children Program, Children’s Health Insurance Program, Medicaid, Medicare).
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On Aug. 27, 2025, the FDA approved 2025–26 formulas of the COVID-19 vaccines: Pfizer/Biontech’s mRNA COVID-19 vaccine (Comirnaty), Moderna’s mRNA COVID-19 vaccines (mNexspike and Spikevax) and Novavax’s adjuvanted COVID-19 vaccine (Nuvaxovid). In May 2025, it was announced that the FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) unanimously voted for the 2025–26 formulas to be monovalent JN.1 lineage compositions. Comirnaty, mNexspike and Spikevax specifically target the LP.8.1 sublineage; this sublineage was preferentially recommended by VRBPAC. Nuvaxovid targets the following circulating JN.1 lineage strains: LP.8.1, NB.1.8.1, XFG, XFC, LF.7 and XEC.
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The HHS also announced the Emergency Use Authorizations (EUAs) for all COVID-19 vaccines have been rescinded. All four vaccines are FDA-approved for active immunization to prevent COVID-19 caused by SARS-CoV-2 in adults ≥ 65 years of age. The vaccines are also approved for certain age ranges of individuals who have at least one underlying condition that puts them at high risk for severe outcomes from COVID-19. The age range for these individuals is six months through 64 years (Spikevax), five years through 64 years (Comirnaty) and 12 years through 64 years (mNexspike, Nuvaxovid).
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The FDA has established a "green list" import alert in an effort to stop potentially dangerous glucagon-like peptide-1 receptor agonists (GLP-1) active pharmaceutical ingredients (APIs) from entering the U.S. market. The intent of the green list is to protect patients who use compounded versions of GLP-1s and will include GLP-1 APIs from facilities that have been inspected/evaluated by the FDA that appear to be in compliance with the FDA’s standards. APIs from other sources not on the green list will be subject to detention without physical examination.
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The FDA is sending thousands of warning letters to pharmaceutical manufacturers for removal of misleading ads; approximately 100 cease-and-desist letters have been issued to those with deceptive ads. The White House has issued a memorandum regarding efforts to address misleading direct-to-consumer (DTC) prescription drug advertising.
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The FDA has announced release of an additional 89 previously unpublished complete response letters (CRLs) or rejection letters associated with pending or withdrawn applications. Moving forward, the Agency will release newly issued CRLs in real-time, promptly following issuance to the application sponsor. Furthermore, when applications are approved, the FDA will release all CRLs associated with that application. The Agency will also be publishing batches of previously issued CRLs for withdrawn or abandoned applications.
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The FDA introduced the Rare Disease Evidence Principles (RDEP) process to provide enhanced efficiency and predictability in the review of investigational therapies for rare genetic diseases with significant unmet medical need. The intent of RDEP is to provide clearer guidance on the types of evidence for investigational products that can be used to demonstrate substantial evidence of effectiveness. Approval under the RDEP process can be based on one adequate and well-controlled study plus robust confirmatory evidence. Application sponsors are able to apply to the process any time before launch of a pivotal trial.
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The Agency’s Center for Drug Evaluation and Research (CDER) has accepted a Letter of Intent for use of liver stiffness measurement by vibration-controlled transient elastography (VCTE) as a reasonably likely surrogate endpoint for all-cause mortality or liver-related events in adults with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis. This proposed biomarker provides a non-invasive method for evaluation of liver stiffness, correlates with liver fibrosis severity, may predict clinical outcomes and may provide a more accessible manner for monitoring progression of disease and treatment response.
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The Agency has begun real-time reporting of adverse event data from the FDA Adverse Event Reporting System (FAERS). The data will be published daily to a public dashboard to streamline the FDA's reporting systems and increase the frequency of reporting to aid in faster identification of safety signals. FAERS is the FDA's primary database for collection of adverse event reports, serious medication errors and product quality complaints for prescription drugs and biologics; HCPs, consumers and manufacturers can submit reports.
A retrospective cohort study published in JAMA Oncology (n=86,632) found that adults with obesity taking GLP-1s had significantly reduced risk of overall cancer, particularly endometrial and ovarian cancers and meningioma, compared to non-GLP-1 users. The study followed a target trial emulation design that used data from electronic health records (EHR) from 2014 to 2024 and investigated incidence of 14 cancer types, including 13 obesity-associated cancers (liver, thyroid, pancreatic, bladder, colorectal, kidney, breast, endometrial, meningioma, upper gastrointestinal, ovarian, multiple myeloma, prostate) and lung cancer. The incidence rate of the 14 cancers was 13.6 per 1,000 person-years for GLP-1 users versus 16.4 per 1,000 person-years for non-users (hazard ratio [HR], 0.83; 95% confidence interval [CI], 0.76 to 0.91; p=0.002). Of note, GLP-1 use was associated with a marginally nonsignificant increased risk of kidney cancer (HR, 1.38; 95% CI, 0.99 to 1.93; p=0.04).
A retrospective cohort study (n=516,052) published in JAMA Ophthalmology evaluated EHR network data from 2006 to 2025 for patients with and without diabetes with prescriptions for GLP-1s, metformin, insulin or sodium-glucose cotransporter 2 inhibitors (SGLT2is). Patients were propensity score matched with other patients without prescriptions for each medication. The researchers found patients who had prescriptions for GLP-1s had a decreased risk of ocular uveitis compared with controls (risk ratio [RR], 0.48; 95% CI, 0.46 to 0.51) with similar findings exhibited for those with and without type 2 diabetes (RR, 0.54 and 0.52, respectively). Prescriptions for SGLT2is were also associated with a decreased uveitis risk versus controls (RR, 0.52; 95% CI, 0.48 to 0.56). Compared with SGLT2is, GLP-1s were associated with a slightly higher uveitis risk (RR, 1.17; 95% CI, 1.04 to 1.32)
A systematic review and meta-analysis of randomized clinical controlled trials (78 trials; n=73,640) published in JAMA Ophthalmology evaluated the number of patients experiencing an ocular adverse event, diabetic retinopathy or nonarteritic anterior ischemic optic neuropathy (NAION) in adults treated with semaglutide. Although semaglutide did not increase or reduce the risk of eye disorders (odds ratio [OR], 1.01; 95% CI, 0.91 to 1.12) or diabetic retinopathy (OR, 1.04; 95% CI, 0.92 to 1.17), treatment with semaglutide was associated with NAION (OR, 3.92; 95% CI, 1.02 to 15.02).
AstraZeneca has announced the launch of FluMist Home, an at-home delivery service for influenza vaccine live, intranasal (FluMist); this product is the first seasonal influenza vaccine approved for self-administration by adults (18 to 49 years of age) or for administration by a parent/caregiver for children (2 to 17 years of age). For the upcoming influenza season, FluMist Home will be available in 34 states; FluMist Home is unavailable in a number of states this season due to local pharmacy laws. FluMist continues to be available in physicians’ offices and pharmacies for HCP administration across the country.
Following a request from the FDA, Intercept has voluntarily withdrawn the primary biliary cholangitis drug obeticholic acid (Ocaliva) from commercialization in the United States. Additionally, a clinical hold is in place from the FDA on all clinical trials conducted under an investigational new drug (IND) application for obeticholic acid. Ocaliva will be unavailable after November 14, 2025.
Unichem has issued a voluntary nationwide recall of one lot of cyclobenzaprine hydrochloride (HCl) 10 mg tablets to the consumer level. The recall is due to mislabeling, as the cyclobenzaprine label was inadvertently placed on a bottle containing meloxicam 7.5 mg tablets.
The FDA has issued a Drug Safety Communication recommending an additional, earlier magnetic resonance imaging (MRI) monitoring prior to the third infusion for patients with Alzheimer’s disease taking lecanemab-irmb (Leqembi); the earlier monitoring is to identify patients with amyloid-related imaging abnormalities with edema (ARIA-E).
The CDC reports, as of Sept. 23, 2025, that there were 1,514 confirmed cases of measles in the United States. Cases include, 409 in individuals less than five years of age, 588 in those five to 19 years of age, 509 in those ≥ 20 years of age and eight with unknown age; 92% of cases are among individuals with unvaccinated or unknown vaccination status. Johns Hopkins' Bloomberg School of Public Health is monitoring the measles outbreak in real-time through their International Vaccine Access Center (IVAC). On Sept. 25, 2025, IVAC reported 1,520 total cases in the United States in 2025.
The CDC has published Perspective on Chagas disease recommending the United States be labeled as endemic or hypoendemic in order to improve efforts around surveillance, research and public health response. Chagas disease, also known as American trypanosomiasis, is caused by the parasitic protozoan Trypanosoma cruzi. The infection can be transmitted through congenital, oral or vectorborne routes. Vectorborne infections are spread through contact with the feces of triatomine insects, also known as kissing bugs.
The American College of Cardiology (ACC)/American Heart Association (AHA) Joint Committee on Clinical Practice Guidelines has published an updated guideline on the prevention, detection, evaluation and management of high blood pressure in adults. This replaces the 2017 version of the guideline.
The American Academy of Pediatrics (AAP) has published an evidence-based immunization schedule for children from birth to 18 years of age. The schedule includes recommendations for influenza, COVID-19 and respiratory syncytial virus (RSV) vaccination, as well as updated recommendations on the pentavalent meningococcal vaccine, the starting age of the human papillomavirus (HPV) vaccine and removal of a hepatitis vaccine that is no longer available. The AAP recommends a COVID-19 vaccine for all children ages six through 23 months of age and recommends a single dose of age-appropriate COVID-19 vaccine for all children and adolescents two through 18 years of age in certain risk groups. For RSV, AAP provides recommendations for immunization of infants younger than eight months as well as for select infants and children eight through 19 months of age at high risk of severe RSV disease.
The American Academy of Family Physicians (AAFP) has also released fall immunization recommendations for respiratory viruses (e.g., COVID-19, RSV, influenza). AAFP recommendations for COVID-19 vaccination in pediatric individuals generally align with AAP recommendations; these COVID-19 vaccine recommendations run contrary to the CDC’s recommendations.
The AAP has published a clinical report on management of opioid dependence and withdrawal. The guidance focuses on children prescribed opioids for acute injuries and chronic conditions and updates a prior 2014 statement.
The CDC has published recommendations from the ACIP for the prevention and control of seasonal influenza with vaccines for the 2025–26 influenza season. Notably, the nasal spray live attenuated influenza vaccine (FluMist) has been approved for self- or caregiver administration and is included as an option in the updated recommendations. The age range for use of the recombinant influenza vaccine Flublok has been expanded from ≥ 18 years of age to at least nine years of age. Furthermore, only single-dose seasonal influenza vaccines that do not contain a thimerosal preservative are recommended.
The CDC has also published recommendations from the June 2025 ACIP meeting regarding the use of clesrovimab-cfor (Enflonsia) for RSV. The long-acting monoclonal antibody is recommended as an alternative to nirsevimab-alip (Beyfortus) for infants less than eight months of age born during or entering their first RSV season whose mothers did not receive an RSV vaccine during pregnancy.
The Global Consensus Statement on the Management of Pregnancy in Inflammatory Bowel Disease (IBD) was published in The American Journal of Gastroenterology. Clinical guidance regarding pharmacotherapy in pregnancy and prior to conception is provided.
The Institute for Clinical and Economic Review (ICER) published a final evidence report on treatments for spinal muscular atrophy (SMA) evaluating the comparative effectiveness of apitegromab (investigational) and the disease-modifying therapies nusinersen (Spinraza), onasemnogene abeparvovec-xioi (Zolgensma) and risdiplam (Evrysdi).
The World Health Organization (WHO) has updated the list of essential medicines to include GLP-1s (semaglutide, dulaglutide and liraglutide) as well as the GLP-1/glucose-dependent insulinotropic polypeptide (GIP) dual receptor agonist (tirzepatide) as medicines for diabetes and obesity.
Drug Approvals
Specialty
Aug. 21, 2025 – donidalorsen (Dawnzera)
- Biologics License Application (BLA) approval; Orphan Drug
- Prekallikrein-directed antisense oligonucleotide
- Indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients ≥ 12 years of age
- Solution for injection: 80 mg/0.8 mL in a single-dose autoinjector
- Recommended dosage is administered subcutaneously (SC) every four weeks; every eight week dosing may also be considered; can be self-administered or administered by a caregiver following proper training
- Approval was based on a randomized, double-blind, placebo-controlled, Phase 3 study (OASIS-HAE; n=90) in which treatment with donidalorsen resulted in a statistically significant reduction in the mean HAE attack rate compared to placebo based on the monthly HAE attack rate from week one to week 25; compared to placebo, the mean attack rate was 81% lower (95% confidence interval [CI], 65 to 89; p<0.001) in the arm that received donidalorsen every four weeks and 55% lower (95% CI, 22 to 74; p=0.004) in the arm that received donidalorsen every eight weeks
- Other FDA-approved agents indicated for prophylaxis of HAE attacks include the oral plasma kallikrein inhibitor berotralstat (Orladeyo; taken once daily) as well as the following injected products which can all be self- or caregiver-administered: the SC plasma kallikrein inhibitor lanadelumab-flyo (Takhzyro) (taken every two to four weeks), the SC human plasma-derived concentrate of C1 esterase inhibitor Haegarda (taken every three or four days), the intravenous (IV) human plasma-derived C1 esterase inhibitor Cinryze (taken every three or four days) and the SC activated factor XII (FXIIa) inhibitor garadacimab-gxii (Andembry) (taken once monthly)
- Dawnzera is available from Ionis
Aug. 29, 2025 – rilzabrutinib (Wayrilz)
- New Drug Application (NDA) approval; Fast Track, Orphan Drug
- Bruton’s tyrosine kinase (BTK) inhibitor
- Indicated for the treatment of adults with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment
- Oral tablets: 400 mg
- Recommended dosage is taken orally twice daily with or without food; in patients who experience gastrointestinal symptoms, taking with food may improve tolerability; tablets should be swallowed whole with a glass of water
- Approval was based on a randomized, double-blind, placebo-controlled, parallel-group, Phase 3 study (LUNA3; n=202) that consisted of a 24-week blinded phase; 64% of rilzabrutinib-treated patients and 32% of patients in the placebo group achieved a platelet count response at 12 weeks and were eligible to continue to the 24-week double-blind phase of the trial; a durable platelet response (platelet count ≥50×10⁹/L for at least two-thirds of at least eight of the last 12 of 24 weeks without rescue therapy) occurred in 23% of rilzabrutinib patients compared with 0% of placebo patients (p<0.0001)
- Wayrilz is the first BTK inhibitor FDA-approved for ITP; other similarly indicated agents for immune thrombocytopenia include oral eltrombopag (Alvaiz, Promacta), oral avatrombopag (Doptelet), SC romiplostim (Nplate) and oral fostamatinib (Tavalisse)
- Wayrilz is available from Genzyme
Sept. 19, 2025 – elamipretide (Forzinity)
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NDA approval; Accelerated Approval, Fast Track, Orphan Drug, Priority Review, Rare Pediatric Disease designation; first FDA-approved therapy for Barth syndrome, an ultra-rare disease
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Mitochondrial cardiolipin binder
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Indicated to improve muscle strength in adult and pediatric patients with Barth syndrome weighing ≥ 30 kg; Accelerated Approval is based on an improvement in knee extensor muscle strength, an intermediate clinical endpoint; therefore, continued approval for this use may require demonstration of benefit in confirmatory clinical trials
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Solution for injection: 280 mg/3.5 mL (80 mg/mL) in single-patient-use vials; vials are discarded eight days after opening
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Recommended dosage is administered SC once daily at the same time each day; patients or caregivers can administer following proper training
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Approval was based on a randomized, double-blind, placebo-controlled, crossover, 28-week trial (TAZPOWER; n=12) and the 168-week, open-label, single-arm extension; during the double-blind portion, elamipretide did not demonstrate superiority to placebo for the primary endpoints assessing distance walked during a 6-minute walk test and total fatigue score on the Barth syndrome symptom assessment; although improvements in the secondary endpoint of knee extensor muscle strength were not observed during the randomized portion of the study, increases were observed during the extension portion of the study (week 168: median change from baseline in muscle strength, 63 newtons)
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Forzinity is a first in class mitochondria-targeted therapeutic; prior to the approval of Forzinity, treatment focused on symptom management and prevention of complications
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Forzinity is expected to be available through a specialty pharmacy by the end of 2025
- 505(b)(2) NDA approval; Camcevi (leuprolide) is also available as a 42 mg injectable emulsion administered SC every six months
- Gonadotropin-releasing hormone (GnRH) agonist
- Indicated for the treatment of adults with advanced prostate cancer
- Injectable emulsion: 21 mg of leuprolide (equivalent to approximately 24 mg leuprolide mesylate) in a single-dose prefilled syringe
- Recommended dosage is administered SC once every three months into the upper or mid-abdominal area; administered by a health care professional (HCP); treatment is typically continued upon development of metastatic castration-resistant prostate cancer
- Camcevi ETM will be available from Foresee with launch timeframe to be determined (TBD)
- 505(b) NDA approval; Orphan Drug; Otezla (apremilast) is also available as an immediate release (IR) tablet (10 mg, 20 mg, 30 mg)
- Phosphodiesterase 4 inhibitor
- Indicated for the treatment of adults with (1) active psoriatic arthritis (PsA), (2) plaque psoriasis who are candidates for phototherapy or systemic therapy and (3) oral ulcers associated with Behçet’s disease; as well as pediatric patients ≥ 6 years of age with (1) active PsA and (2) moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy; in pediatric patients, the XR formulation is indicated for patients weighing ≥ 50 kg; the IR tablet is indicated for patients weighing ≥ 20 kg
- Extended-release (ER) tablets: 75 mg
- Recommended dosage is taken orally and is dependent on patient age and body weight (for pediatric patients); dosage regimens require titration to the maintenance dosage by day six; maintenance dosage for adult indications and pediatric patients ≥ 50 kg is either taken twice daily if using the IR tablet or taken once daily if using the ER tablet; pediatric patients 20 kg to < 50 kg require use of the IR tablets taken twice daily
- Otezla XR is available from Amgen
- BLA approval; Leqembi (lecanemab-irmb) is also available as a solution for IV infusion over approximately one hour administered by an HCP as a weight-based dose once every two weeks for 18 months; after 18 months, treatment can be continued as a once every two week infusion or once every four week maintenance IV infusion
- Amyloid beta-directed antibody
- Indicated for the treatment of Alzheimer’s disease; treatment with Leqembi should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials
- Injection: 360 mg/1.8 mL (200 mg/mL) in a single-dose prefilled Iqlik SC autoinjector
- Recommended maintenance dosage is administered SC in approximately 15 seconds once a week using the Iqlik autoinjector; patients and/or caregivers can administer SC following proper training; patients must start on the IV infusion and continue for 18 months before transitioning to the SC fixed maintenance dosage once weekly
- Boxed warning for amyloid related imaging abnormalities (ARIA)
- Leqembi Iqlik will be available from Biogen/Eisai on Oct. 6, 2025
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505(b)(2) NDA approval; first intravesical drug releasing system (iDRS) to provide extended local delivery of a chemotherapeutic agent into the bladder; Assessment Aid, Breakthrough Therapy, Priority Review, Real-Time Oncology Review (RTOR)
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Nucleoside metabolic inhibitor
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Indicated for the treatment of adult patients with Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors
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Gemcitabine intravesical system: 225 mg; co-packaged with a urinary catheter and stylet used for administration
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Recommended dosage is an intravesical system inserted into the bladder once every three weeks for up to six months (eight doses), followed by once every 12 weeks for up to 18 months (six additional doses), or until persistent or recurrent NMIBC, disease progression or unacceptable toxicity; remove the intravesical system after each three-week indwelling period; product should only be inserted and removed by a trained HCP; patients should drink approximately 1,500 mL of fluids per day during therapy to ensure sufficient urine production for drug release; patients should receive counseling on avoidance of urine contact with skin, voiding sitting on a toilet, etc. during the three week indwelling period for Inlexzo
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Inlexzo is available from Janssen
- BLA approval; Assessment Aid
- Combination of the programmed death receptor-1 (PD-1)-blocking antibody pembrolizumab and the endoglycosidase berahyaluronidase alfa
- Indicated for select patients with (1) melanoma, (2) non-small cell lung cancer, (3) malignant pleural mesothelioma, (4) head and neck squamous cell cancer, (5) urothelial cancer, (6) microsatellite instability-high or mismatch repair deficient cancer, (7) microsatellite instability-high or mismatch repair deficient colorectal cancer, (8) gastric cancer, (9) esophageal cancer, (10) cervical cancer, (11) hepatocellular carcinoma, (12) biliary tract cancer, (13) Merkel cell carcinoma, (14) renal cell carcinoma, (15) endometrial carcinoma, (16) tumor mutational burden-high cancer, (17) cutaneous squamous cell carcinoma and (18) triple-negative breast cancer; consult the prescribing information for specific indications
- Solution for injection: 395 mg pembrolizumab and 4,800 units berahyaluronidase alfa per 2.4 mL and 790 mg pembrolizumab and 9,600 units berahyaluronidase alfa per 4.8 mL in single-dose vials
- Recommended dosage is administered SC into the thigh or abdomen by an HCP; different dosage and administration than IV pembrolizumab (Keytruda); every three week dosing is administered over one minute and every six week dosing is administered over two minutes
- Keytruda Qlex will be available from Merck in late September 2025
Aug. 29, 2025 – denosumab-nxxp (Bildyos)
- BLA approval; denosumab-nxxp (Bildyos) is a biosimilar to reference drug denosumab (Prolia); fifth FDA-approved biosimilar to Prolia (one of which is interchangeable)
- RANK ligand inhibitor
- Indicated for the treatment (1) of postmenopausal women with osteoporosis at high risk for fracture; (2) to increase bone mass in men with osteoporosis at high risk for fracture; (3) of glucocorticoid-induced osteoporosis in men and women at high risk for fracture; (4) to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer and (5) to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer (same indications as Prolia)
- Solution for injection: 60 mg/mL single-dose prefilled syringe (same as Prolia) and 60 mg/mL single-dose vial
- Recommended dosage is given every six months as a SC injection in the upper arm, upper thigh or abdomen administered by an HCP; patients should take calcium and vitamin D daily
- Boxed warning for severe hypocalcemia in patients with advanced kidney disease
- Product is available from Organon
Aug. 29, 2025 – denosumab-nxxp (Bilprevda)
- BLA approval; denosumab-nxxp (Bilprevda) is a biosimilar to reference drug denosumab (Xgeva); fifth FDA-approved biosimilar to Xgeva (one of which is interchangeable)
- RANK ligand inhibitor
- Indicated for (1) prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors; (2) treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity and (3) treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy (same indications as reference drug Xgeva)
- Solution for injection: 120 mg/1.7 mL in a single-dose vial (same as Xgeva)
- Recommended dosage is administered every four weeks SC into the upper arm, upper thigh or abdomen by an HCP; additional doses are given on days eight and 15 for the first month of therapy in patients being treated for giant cell tumor of bone or hypercalcemia of malignancy; calcium and vitamin D should be taken as needed to treat/prevent hypocalcemia
- Product is available from Organon
Sept. 16, 2025 – denosumab-kyqq (Aukelso)
- BLA approval; denosumab-kyqq (Aukelso) is a biosimilar to reference drug denosumab (Xgeva); sixth FDA-approved biosimilar to Xgeva (one of which is interchangeable); Aukelso has received provisional interchangeability designation
- RANK ligand inhibitor
- Indicated for (1) prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors; (2) treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity and (3) treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy (same indications as reference drug Xgeva)
- Solution for injection: 120 mg/1.7 mL in a single-dose vial (same as Xgeva)
- Recommended dosage is administered every four weeks SC into the upper arm, upper thigh or abdomen by an HCP; additional doses are given on days eight and 15 for the first month of therapy in patients being treated for giant cell tumor of bone or hypercalcemia of malignancy; calcium and vitamin D should be taken as needed to treat/prevent hypocalcemia
- Product will be available from Biocon with launch timeframe TBD
Sept. 16, 2025 – denosumab-kyqq (Bosaya)
- BLA approval; denosumab-kyqq (Bosaya) is a biosimilar to reference drug denosumab (Prolia); sixth FDA-approved biosimilar to Prolia (one of which is interchangeable); Bosaya has received provisional interchangeability designation
- RANK ligand inhibitor
- Indicated for the treatment (1) of postmenopausal women with osteoporosis at high risk for fracture; (2) to increase bone mass in men with osteoporosis at high risk for fracture; (3) of glucocorticoid-induced osteoporosis in men and women at high risk for fracture; (4) to increase bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer and (5) to increase bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer (same indications as Prolia)
- Solution for injection: 60 mg/mL single-dose prefilled syringe (same as Prolia)
- Recommended dosage is given every six months as a SC injection in the upper arm, upper thigh or abdomen administered by an HCP; patients should take calcium and vitamin D daily
- Boxed warning for severe hypocalcemia in patients with advanced kidney disease
- Product will be available from Biocon with launch timeframe TBD
Sept. 5, 2025 – von Willebrand factor, recombinant (Vonvendi)
- Takeda; recombinant von Willebrand factor (rVWF); Orphan Drug, Priority Review
- Expanded indication: (1) for routine prophylaxis to reduce the frequency of bleeding episodes in adults with all types of von Willebrand disease (VWD) and (2) on-demand treatment and control of bleeding episodes and perioperative management of bleeding in pediatric patients with VWD; previously, it was indicated for (1) preventative use only in adults with Type 3 VWD, and (2) on-demand treatment and control of bleeding episodes and perioperative management of bleeding in adults
- Administered IV after reconstitution; can be self-administered or administered by a caregiver; recommended dosage and frequency are individualized based on the patient’s body weight, type of bleed, bleed location and severity of the bleeding episode or surgical intervention; appropriate clinical and laboratory measures should also be considered with individualizing the dosage; for prophylactic treatment, the recommended dosage is weight-based and given twice weekly
Sept. 10, 2025 – selumetinib (Koselugo)
- AstraZeneca; kinase inhibitor; Breakthrough Therapy, Orphan Drug; FDA also approved a new oral granule formulation (5 mg and 7.5 mg)
- Expanded indication: to include pediatric patients ≥ 1 year of age with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN); previously, Koselugo capsules were approved for these patients who were ≥ 2 years of age
- Administered orally twice daily with dosing based on body surface area (BSA); the previously approved capsule formulation is swallowed whole; the new granule formulation is sprinkled onto or mixed with soft food
Sept. 12, 2025 – beremagene geperpavec-svdt (Vyjuvek)
- Krystal; herpes-simplex virus type 1 (HSV-1) vector-based gene therapy; FDA also approved application of Vyjuvek in a home setting by an HCP, patient or caregiver
- Expanded indication: to include pediatric patients from birth through adults for the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene; previously, Vyjuvek was approved for this use in patients ≥ 6 months of age
- Administered topically once a week to the selected wound(s) in droplets spaced evenly within the wound (approximately 1 cm-by-1 cm apart); maximum weekly dose is based on patient age (< 3 years of age versus ≥ 3 years of age); product is dosed in plaque forming units (PFU)
Traditional
Aug. 29, 2025 – escitalopram (no trade name)
- 505(b)(2) NDA approval; escitalopram is also available as an oral solution (5 mg/5 mL, 10 mg/mL) (generic) and an oral tablet (5 mg, 10 mg, 20 mg) (Lexapro, generics)
- Selective serotonin reuptake inhibitor (SSRI)
- Indicated for the treatment of (1) major depressive disorder in adults < 65 years of age and pediatric patients ≥ 12 years of age and (2) generalized anxiety disorder in adults < 65 years of age
- Oral capsules: 15 mg
- Recommended dosage is administered orally once daily with or without food; another escitalopram product should be used for initiation, titration, dosages other than 15 mg and discontinuation; this product is not indicated in geriatric patients and is not recommended in patients with hepatic impairment; this 15 mg capsule can be initiated in patients experiencing unfavorable tolerability to a 20 mg dosage
- Product will be available from Almatica with launch timeframe TBD
Sept. 9, 2025 – bimatoprost ophthalmic gel (Zolymbus)
- 505(b)(2) NDA approval; bimatoprost is also approved as a 0.03% ophthalmic solution (generics), a 0.01% ophthalmic solution (Lumigan) and a 10 mcg implant (Durysta), all of which are indicated for reduction of elevated intraocular pressure (IOP) in patients with open-angle glaucoma (OAG) or ocular hypertension (OHT); there is also a 0.03% formulation for hypotrichosis of the eyelashes (Latisse, generics)
- Prostaglandin analog
- Indicated for reduction of elevated IOP in patients with OAG or OHT
- Ophthalmic gel: 0.01% bimatoprost in a single-dose container; container should be thrown away after use even if there is product left in the container
- Recommended dosage is one drop in the affected eye(s) once daily in the evening; can be used concurrently with other topical ophthalmic drugs to lower IOP (separate drug administration by at least five minutes)
- Product will be available from Thea with launch timeframe TBD
Sept. 12, 2025 – bumetanide nasal spray (Enbumyst)
- 505(b)(2) NDA approval; bumetanide is also FDA-approved as an oral tablet (Bumex, generics) and a solution for injection (generic); Enbumyst is the first FDA-approved intranasal loop diuretic
- Loop diuretic
- Indicated for the treatment of edema associated with congestive heart failure, hepatic and renal disease, including nephrotic syndrome in adults
- Nasal spray: 0.5 mg bumetanide per 0.1 mL spray per unit dose nasal spray device; each device is for single use and delivers one spray upon actuation; doses over 0.5 mg will require more than one device; can be substituted at approximately a 1:40 ratio to oral furosemide and a 1:20 ratio to IV furosemide
- Recommended dosage is self or caregiver administered once daily directly into the nose; dosage is individualized based on patient response; if dose requires more than one nasal spray/device, alternate between right and left nostrils; for short term use only, not intended for chronic use and should be replaced with oral diuretics as soon as practical
- Product will be available from Corstasis in the fourth quarter of 2025
Sept. 16, 2025 – lamotrigine oral suspension (Subvenite)
- 505(b)(2) NDA approval; lamotrigine is also FDA-approved as a chewable tablet (Lamictal, generics), an orally disintegrating tablet (Lamictal ODT, generics), an oral tablet (Lamictal, generic) and an extended-release oral tablet (Lamictal XR, generics); Subvenite is the first FDA-approved lamotrigine oral suspension
- Anti-epileptic drug
- Indicated for (1) adjunctive therapy in epilepsy in patients ≥ 2 years of age (partial-onset seizures, primary generalized tonic-clonic (PGTC) seizures, generalized seizures of Lennox-Gastaut syndrome); (2) monotherapy for epilepsy in patients ≥ 16 years of age (conversion to monotherapy in patients with partial-onset seizures who are receiving treatment with carbamazepine, phenytoin, phenobarbital, primidone or valproate as the single antiepileptic drug) and (3) maintenance treatment of bipolar I disorder to delay the time to occurrence of mood episodes in patients treated for acute mood episodes with standard therapy
- Limitations of use: not recommended for the treatment of acute manic or mixed episodes; effectiveness in the acute treatment of mood episodes has not been established
- Oral suspension: 10 mg/mL
- Recommended dosage is based on concurrent medications, indication for use and patient age; taken every other day, once daily, or in two divided doses with or without food with a calibrated measuring device (e.g., oral dosing syringe, oral dosing cup); to avoid an increased risk of rash, the recommended dosages (initial and dose escalations) should not be exceeded
- Boxed warning for serious skin rashes
- Product will be available from OWP later in 2025
Aug. 21, 2025 – evolocumab (Repatha)
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Amgen; proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor
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Expanded indication: indication to reduce the risk of major adverse cardiovascular (CV) events (MACE) (e.g., CV death, myocardial infarction, stroke, unstable angina requiring hospitalization, coronary revascularization) in adults has been expanded to include adults who are at increased risk for these events; Repatha was previously indicated to reduce the risk of MACE in adults with established CV disease
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Expanded indication: as an adjunct to diet and exercise to reduce low-density lipoprotein cholesterol (LDL-C) in (1) adults with hypercholesterolemia; (2) adults and pediatric patients ≥ 10 years of age with heterozygous familial hypercholesterolemia (HeFH) and (3) adults and pediatric patients ≥ 10 years of age with homozygous familial hypercholesterolemia (HoFH); these indications previously stated that Repatha was to be (1) used as an adjunct to diet, alone or in combination with other LDL-C lowering therapies, in adults with primary hyperlipidemia, including HeFH; (2) used as an adjunct to diet and other LDL-C-lowering therapies for pediatric patients ≥ 10 years of age with HeFH and (3) used as an adjunct to other LDL-C-lowering therapies in adults and pediatric patients ≥ 10 years of age with HoFH
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Administered as a SC injection every two weeks or every month; can be self- or caregiver- administered following proper training
Sept. 18, 2025 – ruxolitinib (Opzelura)
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Incyte; Janus kinase (JAK) inhibitor; first FDA-approved topical JAK inhibitor for pediatric atopic dermatitis
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Expanded indication: for pediatric patients ≥ 2 years of age to < 12 years for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised patients whose disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable; previously approved for this indication in adults and pediatric patients ≥ 12 years of age
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Applied topically as a thin layer twice daily to affected areas of up to 20% body surface area (BSA); pediatric patients 2 to 11 years of age should not use more than one 60 gram tube per 2 weeks
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Other indication: topical treatment of nonsegmental vitiligo in adult and pediatric patients ≥ 12 years of age
First generic drug launches
- Natco/Lupin launched a generic dispersible tablet to Actelion’s Tracleer
- Endothelin receptor antagonist; indicated for the treatment of pulmonary arterial hypertension (PAH) (WHO Group 1): (1) in adults to improve exercise ability and to decrease clinical worsening; and (2) in pediatric patients ≥ 3 years of age with idiopathic or congenital PAH to improve pulmonary vascular resistance (PVR), which is expected to result in an improvement in exercise ability
- Recommended dosage is taken orally twice daily for patients > 12 years of age and can be further increased after four weeks for patients > 40 kg; for patients ≤ 12 years of age, dosage is based on body weight and taken twice daily; tablets for oral suspension, or dispersible tablet half, should be dispersed in a minimal amount of water immediately prior to administration
- Annual sales for Tracleer dispersible tablets in 2024 were $12 million
Aug. 28, 2025 – liraglutide injection (Saxenda)
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Teva launched a generic 6 mg/mL solution in 3 mL prefilled, single-patient-use pens to Novo Nordisk’s Saxenda pens in the same presentation
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Glucagon like peptide-1 (GLP-1) receptor agonist; indicated in combination with a reduced-calorie diet and increased physical activity to reduce excess body weight and maintain weight reduction long term in: (1) adults and pediatric patients ≥ 12 years of age with body weight > 60 kg and obesity and (2) adults with overweight in the presence of at least one weight-related comorbid condition
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Recommended dosage is a daily SC injection up-titrated at weekly intervals until the target dose is reached; if pediatric patients do not tolerate an increased dose during dose escalation, the dose may also be reduced to the prior level (dose escalation in these patients may take up to eight weeks)
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Annual sales for Saxenda in 2024 were $217 million
COVID-19 coronavirus disease 2019
mRNA messenger ribonucleic acid
RANK receptor activator of nuclear factor κB
RNA ribonucleic acid
SARS-CoV-2 severe acute respiratory syndrome coronavirus 2
WHO World Health Organization
XR extended-release
Editor-In-Chief: Maryam Tabatabai, PharmD
Executive Editor: Anna Schreck Bird, PharmD
Deputy Editors: Nicole Kjesbo, PharmD, BCPS; Olivia Pane, PharmD, CDCES
All brand names are property of their respective owners.