Trending Topics & Drug Approvals: August 2025 - Prime Therapeutics
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Trending Topics & Drug Approvals: August 2025
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August 29, 2025
Trending Topics
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BARDA research update
The United States (U.S.) Department of Health and Human Services (HHS) has announced that mRNA vaccine development activities under Biomedical Advanced Research and Development Authority (BARDA) will be concluding. This includes termination of 22 mRNA vaccine development investments. Although certain contracts that are in the final stages will complete existing projects, no new mRNA vaccine initiatives will be started. The mRNA projects reviewed for this decision were initiated during the COVID-19 public health emergency (PHE). The HHS reiterates their support for safe and effective vaccines and plans to refocus financial investments in vaccine platforms that continue to be effective for mutating viruses. HHS has stated the decision to terminate the mRNA-based contracts under BARDA was made based on data from a comprehensive review that demonstrated the mRNA vaccines did not effectively provide protection against upper respiratory infections (e.g., COVID-19, influenza). Other mRNA technology platforms covered under BARDA are unaffected by this decision. BARDA’s focus will be platforms with stronger safety signals and transparent data practices in both the clinical and manufacturing settings. The intent is to phase out funding initiated during the COVID-19 PHE that does not meet current scientific standards. New platforms explored will be evidence-based and may include whole-virus vaccines or novel platforms. Following the release of the HHS announcement, physician groups issued a statement in opposition of the decision to remove mRNA vaccine research funding for respiratory viruses under BARDA.New FDA program
The U.S. Food and Drug Administration (FDA) has announced the new PreCheck program to increase U.S. drug manufacturing. The intent of the new program is to facilitate additional domestic manufacturing sites through increased regulatory predictability. Currently, more than half of U.S. drug products are manufactured overseas. Furthermore, only 11% of active pharmaceutical ingredient (API) manufacturers are based in the United States. PreCheck was developed in response to Executive Order 14293: "Regulatory Relief to Promote Domestic Production of Critical Medicines" that directs the FDA to streamline review of U.S. drug manufacturing. The PreCheck program will involve removal of unnecessary regulatory requirements and maximization of review timeframes. The program will be comprised of a two-phase approach: (1) Facility Readiness Phase and (2) Application Submission Phase. The Facility Readiness Phase allows for more frequent communication with the FDA during facility design and construction, as well as prior to drug production. Manufacturers are encouraged to submit a facility-specific Type V Drug Master File (DMF), which can be incorporated into a drug application as appropriate. The Application Phase will streamline the Chemistry, Manufacturing and Controls (CMC) section of an application through pre-application meetings with the FDA as well as allow for early feedback. The upcoming "Onshoring Manufacturing of Drugs and Biological Products" meeting on September 30, 2025, will include a presentation on FDA PreCheck as well as stakeholder discussions.A research letter published in JAMA Ophthalmology discusses an association between nonarteritic anterior ischemic optic neuropathy (NAION) and use of glucagon-like peptide-1 receptor agonists (GLP-1s) in Medicare enrollees ≥ 65 years of age with type 2 diabetes mellitus (T2DM) (n=3,845,171). The observational cohort study found 0.2% (n=7,660) of patients developed NAION over a median follow-up of 3.7 years equating to a hazard ratio (HR) of 1.15 for use of any GLP-1 compared with other second-line diabetes medications. The median time from initiation of a GLP-1 and incidence of NAION was 3.3 years.
An article published in JAMA Internal Medicine addresses strategies for integrating diet and physical activity for patients taking GLP-1s, with a focus on managing gastrointestinal symptoms and supporting muscle and nutrient preservation during treatment. To integrate nutrition and physical activity for these patients, the MEAL mnemonic can be used: muscle maintenance (adequate protein intake, physical activity), energy balance (smaller, nutrient-dense meals preferred over calorie restriction; whole grains and healthy fats), avoid side effects (constipation management: fiber, hydration and over-the-counter laxatives; nausea management: avoid fried foods/carbonated beverages; gastroesophageal reflux management: smaller portions, no lying down for two to three hours after meals, limit high-fat/irritating spices), liquid intake (consume more than two to three liters of water per day as well as foods rich in water/electrolytes). Additional details are provided on management of micronutrients deficiencies, physical activity recommendations and weight loss maintenance following medication discontinuation.
A nationally representative cross-sectional study (n=25,847) published in JAMA Network Open evaluated data from pediatric participants aged 2 to 18 years old from the National Health and Nutrition Examination Survey (NHANES) from 2008 through 2023. The U.S. survey assessed health and nutrition through questionnaires, laboratory tests and physical examinations and found the prevalence of pediatric obesity increased from 2008 to 2023. The prevalence of extremely severe obesity in U.S. children, defined as body mass index (BMI) ≥ 160% of 95th percentile, increased by 253.1% (from 0.32% in 2008 to 1.13% in 2023). From 2020 to 2023, the prevalence of extremely severe obesity was highest among adolescents aged 16 to 18 years (1.99%; 95% confidence interval [CI], 1.31% to 2.99%) and non-Hispanic Black participants (2.04%; 95% CI, 1.45% to 2.86%). Additionally, this classification of obesity was associated with higher odds of metabolic complications, including metabolic dysfunction–associated steatotic liver disease (MASLD) (odds ratio [OR], 6.74; 95% CI, 3.3 to 15.75), prediabetes or diabetes (OR, 4.94; 95% CI, 3.41 to 7.14), severe insulin resistance (OR, 8.05; 95% CI, 3.7 to 17.02) and metabolic syndrome (OR, 1.99; 95% CI, 1.45 to 2.73).
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The FDA has suspended the biologics license for Valneva’s Chikungunya Vaccine, Live (Ixchiq) following serious adverse events in which the vaccine caused chikungunya-like illness in vaccine recipients. One death due to encephalitis has been attributed to the vaccine and more than 20 serious adverse events have been reported including 21 hospitalizations and three deaths. Additionally, the clinical benefit of the vaccine has not been demonstrated in confirmatory clinical studies (a requirement of the vaccine’s November 2023 Accelerated Approval). In May 2025, the Agency recommended a pause in use of the vaccine for individuals ≥ 60 years of age. In early August 2025, the pause was removed and label updates were made to the prescribing information revising the indication to state the vaccine is for the prevention of disease caused by chikungunya virus (CHIKV) in individuals ≥ 18 years of age who are at high risk of exposure to CHIKV; previously, it was indicated for those at increased risk. A limitation of use was also added at that time stating vaccination with Ixchiq is not advisable for most U.S. travelers; as for most travelers, the risk of exposure to CHIKV is low. However, following the label updates, the FDA subsequently suspended the biologics license for the vaccine on Aug. 22, 2025. The suspension takes effect immediately requiring Valneva to stop shipment and sale of Ixchiq in the United States. The safety concerns with Ixchiq were first reported in the May edition of Prime's Trending Topics & Drug Approvals.
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The FDA has provided an update to the November 2024 FDA Safety Communication regarding the serious risk of hematological malignancy after elivaldogene autotemcel (Skysona) treatment. Following a review of the clinical trial data, the Agency has required prescribing information updates to the Boxed Warning, Indications and Usage, Warnings and Precautions and Adverse Reactions sections to include new safety information on the increased risk of hematologic malignancy; the revisions include restricting the indication to patients without an available human leukocyte antigen (HLA)-matched allogeneic hematopoietic stem cell (allo-HSC) donor. As a result, the single-dose gene therapy should only be used in cerebral adrenoleukodystrophy (CALD) patients without suitable alternative treatment options due to the increased risk of hematologic malignancy.
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The FDA is requiring safety labeling changes to all opioid pain medications regarding risks associated with long-term use in order to assist with informed treatment decision making. The safety communication follows a meeting of the Drug Safety and Risk Management Advisory Committee and the Anesthetic and Analgesic Drug Products Advisory Committee in May 2025 that discussed serious risks for users of opioids over extended periods of time, including the potential for misuse, addiction and fatal/non-fatal overdoses. The updated labeling will include data from two large observational studies, and the FDA is requiring an additional prospective randomized controlled clinical trial to evaluate the benefits versus risk of long-term opioid use.
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Shortages of stimulants for the treatment of attention-deficit/hyperactivity disorder (ADHD) continue to be reported. Availability of generic methylphenidate hydrochloride (HCl) extended-release (ER) tablets is variable depending on the manufacturer. Brand name Relexxii from Vertical has variable availability; brand name Concerta from Janssen is currently available. Generic methylphenidate ER film is available; however, brand name Daytrana from Noven is unavailable due to a shortage of an active ingredient. Generic amphetamine aspartate monohydrate, amphetamine sulfate, dextroamphetamine saccharate and dextroamphetamine sulfate immediate release (IR) tablet shortages are also being reported; all strengths of brand-name Adderall IR tablets from Teva are available. Shortages of the generic version of Vyvanse, lisdexamfetamine dimesylate capsules and chewable tablets, persist. Brand name Vyvanse capsules and chewable tablets remain available from Takeda.
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The FDA is continuing to report shortages of penicillin G benzathine (Bicillin L-A). The 1,200,000 IU/2 mL strength has limited availability, the 2,400,000 IU/4 mL strength is unavailable due to a manufacturing delay and the 600,000 IU/mL strength is unavailable with next delivery in October 2025 and estimated recovery of December 2025. Non-FDA approved benzathine benzylpenicillin tetrahydrate (Lentocilin), from Portugal, and benzathine benzylpenicillin (Extencilline), manufactured in Italy and marketed in France, remain available through temporary importation. The shortage of Bicillin L-A coincides with a voluntary recall of certain lots of Bicillin L-A by King (a subsidiary of Pfizer). As a result, the Centers for Disease Control and Prevention (CDC) recommends prioritization of Bicillin L-A for treating women with syphilis during pregnancy, as penicillin is the only recommended treatment for these patients and babies with congenital syphilis. Additional priority actions for management of the shortage/recall are provided from the CDC.
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The FDA’s new Commissioner’s National Priority Voucher (CNPV) pilot program is now accepting applications. The program intends to shorten the FDA review time for approval of drugs and biologics from 10 to 12 months to one to two months after final drug application submission. For more information on the pilot program, please visit the June 2025 Trending Topics & Drug Approvals.
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An observational cohort study published in Pediatrics evaluated healthy term infants during the 2023-24 respiratory syncytial virus (RSV) season at Kaiser Permanente Northern California. The study assessed the effectiveness of nirsevimab (Beyfortus) against RSV and RSV-related events in infants (n=31,900), 49.1% (n=15,647) of whom received nirsevimab. Estimated treatment effectiveness of nirsevimab was 87.2% (95% CI, 81.7% to 91.1%; p<0.001) against first episode of polymerase chain reaction (PCR)-confirmed RSV lower respiratory tract disease (LRTD; primary outcome) with 35 episodes in infants who received nirsevimab compared with 462 in infants who did not receive nirsevimab. The estimated treatment effectiveness of nirsevimab for PCR-confirmed RSV (secondary outcome) was 71% (95% CI, 65.3% to 75.8%; p<0.001). Post hoc analysis demonstrated nirsevimab was associated with an adjusted effectiveness of 98% (95% CI, 85.1% to 99.7%; p<0.0001) against hospitalized RSV LRTD compared to no receipt of nirsevimab. Furthermore, infants with RSV LRTD who received nirsevimab had fewer medical encounters (adjusted means difference, -0.86; p=0.001) as well as lower odds of hospitalization (adjusted OR, 0.11; 95% CI, 0.01 to 0.85; p=0.035) than infants who had not received nirsevimab.
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The manufacturer of RSV drug palivizumab (Synagis) has announced the product will be voluntarily discontinued. As of Dec. 31, 2025, Synagis will no longer be available. The FDA has also reported discontinuation of the product. Synagis is a monoclonal antibody indicated for select pediatric patients for the prevention of serious LRTD caused by RSV.
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The CDC reports, as of Aug. 26, 2025, that there were 1,408 confirmed cases of measles in the United States. Cases include, 393 in individuals < 5 years of age, 531 in those 5 to 19 years of age, 477 in those ≥ 20 years of age and 7 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 Aug. 28, 2025, IVAC reported 1,401 total cases in the United States in 2025.
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Recommendations from the June 25–26, 2025 Advisory Committee on Immunization Practices (ACIP) meeting on use of clesrovimab (Enflonsia) were adopted by the CDC director on Aug. 4, 2025. Infants aged < 8 months born during or entering their first RSV season who are not protected by maternal vaccination are recommended to receive one dose of clesrovimab. The CDC states there is not a preferential recommendation of nirsevimab (Beyfortus) or Enflonsia. Seasonal influenza vaccine recommendations from the ACIP meeting have also been adopted as official CDC recommendations: (1) routine annual influenza vaccination is recommended for persons aged ≥ 6 months who do not have contraindications for the 2025-26 influenza season, and (2) children ≤ 18 years of age, pregnant women and all adults are recommended to receive seasonal influenza vaccination with only single-dose thimerosal-free formulations.
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A joint statement has been issued by the American Academy of Family Physicians, American Academy of Pediatrics (AAP), American College of Physicians, American Geriatrics Society, American Medical Association, American Osteopathic Association, Infectious Diseases Society of America and National Medical Association in response to an email from ACIP that liaison organizations will be excluded from the scientific review process for informing vaccine recommendations. The organizations are strongly urging the Administration to reconsider their exclusion from participation in the ACIP vaccine review process.
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The AAP released influenza vaccine recommendations for the 2025-26 season. An influenza vaccine is recommended for all children ≥ 6 months of age. The United States experienced a high-severity 2024-25 influenza season for persons of all ages. According to the CDC, 253 influenza-related pediatric deaths had occurred through late June 2025 with 43% occurring in children that did not have a high-risk medical condition. Any licensed influenza vaccine appropriate for age and health status can be given, as soon as possible in the season but ideally by the end of October. There is no preference for one product or formulation; options include inactivated influenza vaccines (IIV) or recombinant influenza vaccines (RIV) as well as live attenuated influenza vaccines (LAIV) (e.g., nasal spray). LAIV should not be used for immunocompromised persons or those with certain chronic medical conditions. Children 6 months to 8 years of age should receive two doses (given four weeks apart) if this is the first time receiving vaccination for influenza or if they have only received one dose of influenza vaccine ever before July 1.
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The Endocrine Society and European Society of Endocrinology have published a clinical practice guideline for management of preexisting diabetes and pregnancy. Recommendations are provided for pregnancy screening, discontinuation of GLP-1s before conception, use of insulin and metformin in pregnant individuals with T2DM, use of insulin pumps in patients with type 1 diabetes mellitus (T1DM) and glucose monitoring.
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The Endocrine Society has issued a clinical practice guideline for primary aldosteronism which is a primary adrenal disorder resulting in excessive aldosterone production by the adrenal glands. This can lead to hypertension and an increased risk of cardiovascular complications. Based on currently available literature, recommendations for the diagnosis and treatment of primary aldosteronism are provided.
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The American Academy of Otolaryngology–Head and Neck Surgery Foundation has published an updated clinical practice guideline on adult sinusitis. This guideline replaces the 2015 version and provides 14 recommendations on diagnosis of rhinosinusitis, appropriate use of ancillary tests and the judicious use of systemic and topical therapy.
Drug Approvals
Specialty
July 28, 2025 – sepiapterin (Sephience)
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New Drug Application (NDA) approval; Orphan Drug
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Phenylalanine hydroxylase (PAH) activator
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Indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients ≥ 1 month of age with sepiapterin-responsive phenylketonuria (PKU); to be used in conjunction with a phenylalanine (Phe)-restricted diet
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Oral powder: 250 mg or 1,000 mg in a unit-dose packet
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Recommended dosage is administered orally once daily with food at a weight-based dose depending on the patient’s age; following treatment initiation, blood Phe levels should be checked to determine response to therapy within two weeks; if blood Phe levels do not decrease, the dosage may be titrated incrementally based on blood Phe levels to a maximum daily weight-based dosage (60 mg/kg); Sephience should be discontinued in patients whose blood Phe does not decrease following two weeks of treatment at the maximum daily dosage; patients receiving a dose < 1,000 mg should have the powder mixed with an appropriate volume of water or apple juice to result in a 25 mg/mL concentration; for patients receiving a dose of ≥ 1,000 mg, packet contents should be mixed with an appropriate volume of water, apple juice, strawberry jam or applesauce to yield a soft food mixture
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Approval was based on a randomized, double-blind, placebo-controlled Phase 3 study (n=98; APHENITY trial) that demonstrated a 63% mean reduction in blood Phe concentrations from baseline after six weeks of sepiapterin compared with placebo which exhibited a 1% increase from baseline (treatment difference, -64%; p<0.0001); patients enrolled in this part of the study were predetermined to be sepiapterin-responsive
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Other Food and Drug Administration (FDA)-approved treatments for PKU include sapropterin (Kuvan, Javygtor) and pegvaliase-pqpz (Palynziq); Kuvan and Javygtor are also PAH activators supplied as oral tablets and powder for oral solution indicated to reduce blood Phe levels in adult and pediatric patients ≥ 1 month of age with HPA due to tetrahydrobiopterin (BH4)-responsive PKU; Palynziq is a Phe-metabolizing enzyme administered subcutaneously (SC) to reduce blood Phe concentrations in adults with PKU who have uncontrolled blood Phe concentrations > 600 micromol/L on existing management
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Sephience is available from PTC Therapeutics
Aug. 6, 2025 – dordaviprone (Modeyso)
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NDA approval; Accelerated Approval, Assessment Aid, Fast Track, Orphan Drug, Priority Review, Rare Pediatric Disease
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Protease activator
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Indicated for adult and pediatric patients ≥ 1 year of age with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy; the Accelerated Approval is based on response rate and duration of response; therefore, continued approval for this use may require demonstration of benefit in confirmatory clinical trials
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Oral capsule: 125 mg
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Recommended dosage for adults is a fixed dose taken orally once weekly; for pediatric patients, the recommended dosage is based on body weight taken once weekly; Modeyso should be taken on an empty stomach (at least one hour before or three hours after food intake) and continued until disease progression or unacceptable toxicity
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Approval was based on an integrated efficacy population of 50 patients (pediatric, n=4; adult, n=46) conducted in the United States; patients were enrolled across four clinical trials or an expanded access protocol; the overall response rate was 20% (95% confidence interval [CI], 10 to 33.7) with a median duration of response of 11.2 months (95% CI, 3.8 to not reached)
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Approval marks the first FDA-approved systemic therapy for H3 K27M-mutant diffuse midline glioma
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Modeyso is available from Jazz
Aug. 8, 2025 – zongertinib (Hernexeos)
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NDA approval; Accelerated Approval, Assessment Aid, Breakthrough Therapy, Fast Track, Priority Review, Real-Time Oncology Review (RTOR) pilot program; the FDA also approved a companion diagnostic device, Life Technologies Corporation’s Oncomine Dx Target Test, for detection of HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations in patients with non-squamous non-small cell lung cancer (NSCLC)
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Kinase inhibitor
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Indicated for adults with unresectable or metastatic non-squamous NSCLC whose tumors have HER2 (ERBB2) TKD activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy; the Accelerated Approval is based on objective response rate (ORR) and duration of response; therefore, continued approval for this use may require demonstration of benefit in a confirmatory clinical trial
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Oral tablets: 60 mg
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Recommended dosage is based on body weight (< 90 kg, ≥ 90 kg) taken orally once daily with or without food until disease progression or unacceptable toxicity
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Approval was based on results from a single arm, open-label, multi-center, multi-cohort trial (Beamion LUNG-1; n=75) that demonstrated a confirmed ORR of 71% (95% CI, 60 to 80); the median duration of response was 14.1 months (95% CI, 6.9 to not evaluable) and the median progression-free survival was 12.4 months (95% CI, 8.2 to not evaluable)
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According to the National Comprehensive Cancer Network (NCCN) NSCLC guideline, zongertinib is a preferred subsequent therapy option for HER2 (ERBB2) mutation NSCLC with progression following systemic therapy for squamous cell carcinoma or adenocarcinoma; fam-trastuzumab deruxtecan-nxki (Enhertu) is also a preferred subsequent therapy option in this setting
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Hernexeos is available from Boehringer Ingelheim
Aug. 12, 2025 – brensocatib (Brinsupri)
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NDA approval; Priority Review; first FDA-approved treatment for non-cystic fibrosis bronchiectasis (NCFB)
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Dipeptidyl peptidase 1 (DPP1) inhibitor
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Indicated for the treatment of NCFB in adult and pediatric patients ≥ 12 years of age
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Oral tablets: 10 mg and 25 mg
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Recommended dosage is one tablet orally once daily with or without food
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Approval was based on two randomized, double-blind, placebo-controlled, parallel-group, multicenter, multinational clinical trials (ASPEN and WILLOW); the Phase 3 ASPEN trial (n=1,680 adults; n=41 adolescents) was 52 weeks and demonstrated a statistically significant reduction in the annualized rate of adjudicated pulmonary exacerbations with both doses of brensocatib compared to placebo (10 mg dose: rate ratio, 0.79; 95% CI, 0.68 to 0.92; adjusted p=0.004; 25 mg dose: rate ratio, 0.81; 95% CI, 0.69 to 0.94; adjusted p=0.005); the 24-week, Phase 2 WILLOW trial (n=256 adults) demonstrated the time to first pulmonary exacerbation was longer for patients receiving brensocatib compared to placebo (adjusted hazard ratio [HR] for 10 mg dose, 0.58; 95% CI, 0.35 to 0.95; p=0.03; HR for 25 mg dose, 0.62; 95% CI, 0.38 to 0.99; p=0.046)
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First-in-class DPP1 inhibitor that blocks the activation of neutrophil serine proteases during the process of neutrophil maturation thereby targeting the pathogenesis of NCFB; prior to approval of brensocatib, management of bronchiectasis relied on antibiotics to treat infections, expectorants to thin the mucus and bronchodilators to open airways; other modalities include chest physical therapy and hydration; the majority of bronchiectasis cases in adults are NCFB
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Brinsupri is available from Insmed through a comprehensive specialty pharmacy network
Aug. 14, 2025 – zopapogene imadenovec-drba (Papzimeos)
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Biologics License Application (BLA) approval; Breakthrough Therapy, Orphan Drug, Priority Review; first FDA-approved treatment for adults with recurrent respiratory papillomatosis (RRP)
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Non-replicating adenoviral vector-based immunotherapy
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Indicated for the treatment of adults with RRP
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Suspension for SC injection: 5x10¹¹ particle units (PU) in a single-dose vial in an extractable volume of 1 mL
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Recommended dosage is 5x10¹¹ PU per injection administered SC by a health care professional (HCP) four times over a 12-week interval; before the initial administration, a surgical debulking of visible papilloma should be performed to establish minimal residual disease; to maintain minimal residual disease during treatment, visible papilloma should be removed, if present, prior to the third and fourth administrations; the second injection is given two weeks after the initial administration (no less than 11 days after the initial administration) and the third and fourth injections should occur six weeks and 12 weeks, respectively, after initial administration; the lateral regions of the upper arm and thigh should be used for SC injection
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Approval was based on a single-arm, open-label trial assessing adults (n=38) who required three or more surgeries in the 12 months prior for RRP; the primary endpoint of a complete response (defined as no surgical interventions for RRP in the 12 months following treatment) was achieved by 51% of patients; furthermore, of the 18 complete responders, 15 had a continued complete response at 24 months (complete response rate of 43% at two years)
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Papzimeos stimulates an immune response against cells infected with human papillomavirus (HPV) 6 or 11; the rare, chronic disease is associated with significant morbidity (e.g., voice changes, difficulty breathing, airway obstruction); repeated surgical procedures have historically been used to address the benign tumors that grow in the respiratory tract due to persistent HPV 6 or 11
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Papzimeos will be available from Precigen with launch timeframe to be determined (TBD)
None
None
July 23, 2025 – apremilast (Otezla)
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Amgen; phosphodiesterase 4 (PDE4) inhibitor
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Expanded indication: active psoriatic arthritis indication expanded to include pediatric patients ≥ 6 years of age and weighing ≥ 20 kg; previously, only indicated for adults with active psoriatic arthritis
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Administered as an oral maintenance dose based on body weight (20 kg to < 50 kg, ≥ 50 kg) taken twice daily, following an initial titration to reduce gastrointestinal (GI) symptoms
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Other indications are detailed in the product label
July 24, 2025 – avatrombopag (Doptelet)
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Sobi; thrombopoietin receptor agonist; in conjunction with the expanded indication, the FDA approved a new oral granule formulation (Doptelet Sprinkle) for the treatment of thrombocytopenia in pediatric patients 1 to < 6 years of age with persistent or chronic immune thrombocytopenia (ITP; Orphan Drug designation); the tablet and granule formulations are not substitutable on a mg-to-mg basis
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Expanded indication: to include pediatric patients ≥ 1 year of age with persistent or chronic ITP who have had an insufficient response to a previous treatment; previously, only indicated for thrombocytopenia in adults with chronic ITP who have had an insufficient response to a previous treatment
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Administered orally with pediatric patients ≥ 6 years of age initiating therapy as one tablet once daily; for pediatric patients 1 to < 6 years of age, therapy is initiated with Doptelet Sprinkle oral granules (contents of one capsule) orally once daily; following this initial dosing, adjust the dose or frequency of dosing to maintain platelet count ≥ 50 × 10⁹/L; Doptelet Sprinkle capsules should be opened and the oral granule contents mixed with soft food or liquid and consumed immediately after mixing (capsules should not be swallowed whole); the tablet formulation is the approved dosage form for patients ≥ 6 years of age
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Other indication: thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure
July 25, 2025 – lonapegsomatropin-tcgd (Skytrofa)
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Ascendis; human growth hormone
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New indication: for the replacement of endogenous growth hormone in adults with growth hormone deficiency (GHD)
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Administered as a SC injection once weekly with the recommended starting dosage based on age and concomitant use of oral estrogen; the dose can be increased monthly to a higher strength cartridge based on clinical response and/or insulin-like growth factor-1 (IGF-1) concentration drawn four to five days after the prior dose; the dose can be decreased to a lower strength cartridge as needed based on adverse reactions or a weekly average IGF-1 concentration above the age- and sex-specific normal range
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Other indication: treatment of pediatric patients ≥ 1 year of age who weigh ≥ 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone
July 28, 2025 – bedaquiline (Sirturo)
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Janssen; diarylquinoline antimycobacterial drug
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Expanded indication: to include pediatric patients 2 to < 5 years of age and weighing ≥ 8 kg for the treatment of pulmonary tuberculosis (TB) due to Mycobacterium tuberculosis resistant to at least rifampin and isoniazid as part of combination therapy; previously, only indicated for this use in adult and pediatric patients (≥ 5 years of age and weighing ≥ 15 kg)
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Limitation of use: Sirturo should not be used for the treatment of latent, extra-pulmonary or drug-sensitive TB or for the treatment of infections caused by nontuberculous mycobacteria
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Administered orally with food for a total duration of treatment of 24 weeks (once daily for weeks one and two, then three times per week for weeks three to 24); in pediatric patients (≥ 2 years of age and weighing ≥ 8 kg) dosage is based on body weight; Sirturo should be administered by directly observed therapy; tablets are functionally scored and can be split, or tablets can be dispersed in water and administered in water or further mixed with a beverage or soft food; alternatively, tablets can be crushed and mixed with soft food
July 28, 2025 – pegcetacoplan (Empaveli)
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Apellis; complement inhibitor
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New indication: for the treatment of adult and pediatric patients ≥ 12 years of age with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria
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Administered as a fixed dose SC injection twice weekly for adults; for pediatric patients ≥ 12 years of age, the dosage is dependent upon body weight (< 35 kg, 35 kg to < 50 kg, ≥ 50 kg) and is given SC twice weekly; can be administered via a commercially available infusion pump or with the Empaveli Injector, which is a single-use, disposable on-body injector; patients or caregivers may administer after proper training by an HCP
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Other indication: for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH)
July 29, 2025 – tocilizumab-anoh (Avtozma)
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Celltrion; interleukin-6 (IL-6) receptor antagonist; only interchangeable biosimilar to tocilizumab (Actemra)
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New indication: adults and pediatric patients ≥ 2 years of age with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS)
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Administered by an HCP as a weight-based intravenous (IV) infusion for CRS with dosage dependent on if patient body weight is < 30 kg or ≥ 30 kg; given alone or in combination with corticosteroids; if clinical improvement does not occur following the first dose, up to three additional doses of Avtozma can be administered (with an interval of ≥ 8 hours between consecutive doses); SC administration is not an approved route for CRS
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Other indications are detailed in the product label; does not carry indications for systemic sclerosis-associated interstitial lung disease or pediatric patients with COVID-19 that reference drug Actemra carries
July 31, 2025 – concizumab-mtci (Alhemo)
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Novo Nordisk; tissue factor pathway inhibitor (TFPI) antagonist
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Expanded indication: for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ≥ 12 years of age with hemophilia A (congenital factor VIII deficiency) without FVIII inhibitors and hemophilia B (congenital factor IX deficiency) without FIX inhibitors; previously, only indicated for these same patients with factor inhibitors
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Administered as a SC injection with the same recommended dosage for patients with or without factor inhibitors; administered as a weight-based loading dose on day one, followed by a weight-based maintenance dose once daily beginning on day two continued until individualization of maintenance dose; four weeks after starting therapy, measure plasma levels of the drug with an Enzyme-Linked Immunosorbent Assay (ELISA) before administration of next scheduled dose using an FDA-authorized test in order to dose optimize; can be self-administered or administered by a caregiver after appropriate training
Aug. 1, 2025 – dasatinib (Phyrago)
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Handa; kinase inhibitor
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New indications: pediatric patients ≥ 1 year of age with (1) Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML) in chronic phase and (2) newly diagnosed Ph+ acute lymphoblastic leukemia (ALL) in combination with chemotherapy
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Recommended pediatric starting dose for chronic phase CML and ALL is based on body weight and is taken orally once daily with or without food; the dose should be recalculated every three months based on changes in body weight, or more often if needed
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Other indications are detailed in the product label
Aug. 8, 2025 – tocilizumab (Actemra)
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Genentech; IL-6 receptor antagonist; with the approval of this expanded indication on August 8, 2025, the FDA has revoked the previous Emergency Use Authorization (EUA) for this same use that was issued on June 24, 2021
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Expanded indication: pediatric patients ≥ 2 years of age who are hospitalized with COVID-19 and who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO); previously, only indicated for this use in adults and EUA for pediatric patients
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Administered as an IV infusion by an HCP over one hour based on a weight-based dose determined by if the patient is < 30 kg or ≥ 30 kg; if clinical presentation worsens or does not improve following the first dose, one additional infusion can be administered ≥ 8 hours after the initial infusion; SC administration is not approved for this indication
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Other indications are detailed in the product label
Traditional
July 23, 2025 – delgocitinib (Anzupgo)
- NDA approval; Anzupgo is the first FDA-approved treatment for moderate to severe chronic hand eczema
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Janus kinase (JAK) inhibitor
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Indicated for the topical treatment of moderate to severe chronic hand eczema (CHE) in adults who have had an inadequate response to, or for whom topical corticosteroids are not advisable
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Topical cream: 20 mg/gram
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Recommended dosage is to apply twice daily to skin of the affected areas only on the hands and wrists; do not use more than 30 grams per two weeks or 60 grams per month
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Approval was based two randomized, double-blind, vehicle-controlled 16-week trials (n=960; DELTA 1 and 2) that evaluated the primary efficacy endpoint of the proportion of subjects who achieved Investigator’s Global Assessment for chronic hand eczema (IGA-CHE) score treatment success (IGA-CHE TS) at week 16, defined as a score of 0 (clear) or 1 (almost clear) with at least a two-point improvement from baseline; in both trials, a significantly greater proportion of patients who received delgocitinib achieved the primary endpoint compared to vehicle (treatment difference was 10% for DELTA 1 and 22% for DELTA 2; p≤0.0055 for both)
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Atopic dermatitis (AD) is a type of eczema; other similar FDA-approved topical treatments include the JAK inhibitor ruxolitinib (Opzelura) indicated for select patients ≥ 12 years of age with mild to moderate AD, the phosphodiesterase 4 inhibitor crisaborole (Eucrisa) which is also indicated for mild to moderate AD (≥ 3 months of age) and the aryl hydrocarbon receptor agonist tapinarof (Vtama) indicated for AD (≥ 2 years of age)
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Anzupgo is available from Leo
July 31, 2025 – aceclidine (Vizz)
- NDA approval; first FDA-approved aceclidine-based eye drop for the treatment of presbyopia in adults; duration of action up to 10 hours
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Cholinergic agonist
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Indicated for the treatment of presbyopia in adults
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Ophthalmic solution: aceclidine 1.44% in a single-dose vial
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Recommended dosage of one drop instilled in each eye; wait two minutes and instill a second drop in each eye once daily
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Approval was based on two randomized, double-masked, controlled Phase 3 trials (n=466; CLARITY-1 and -2) that evaluated the primary endpoint of gaining three lines or more in distance corrected near visual acuity at 40 cm, without loss of one line or more of distance corrected, distance visual acuity at 4 meters at day one, hour three; in CLARITY-1, 65% of patients treated with aceclidine met the primary endpoint compared with 12% of patients on brimonidine (p<0.01); in CLARITY-2, 71% of patients in the aceclidine arm achieved the primary endpoint compared with 8% in the vehicle group (p<0.01)
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Other FDA-approved treatments for presbyopia include pilocarpine products (Vuity and Qlosi), which are also cholinergic agonists; Vuity is administered as one drop instilled in each eye once daily (an additional dose in each eye can be given three to six hours after the first dose); Qlosi is administered as one drop instilled in each eye once daily (an additional dose in each eye can be given two to three hours after the first dose for a duration of effect of up to eight hours)
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Vizz will be available from Lenz in the middle of Q4 2025
July 23, 2025 – ramipril oral solution (Vostally)
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505(b)(2) NDA approval; ramipril is also available as an oral capsule (Altace, generics)
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Angiotensin converting enzyme (ACE) inhibitor
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Indicated (1) for the treatment of hypertension in adults, to lower blood pressure; lowering blood pressure reduces the risk of fatal and nonfatal cardiovascular (CV) events, primarily strokes and myocardial infarctions (MI), (2) in patients ≥ 55 years of age at high risk of developing a major CV event to reduce the risk of MI, stroke or death from CV causes and (3) in adults with post-MI heart failure (HF) to reduce the risk of CV death and hospitalization for HF
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Oral solution: 1 mg/mL
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Recommended starting dosage is taken orally once or twice daily with dosage dependent on indication; titrate based on response and tolerability to target or maintenance dose
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Boxed warning for fetal toxicity
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Product will be available from Metacel/Rosemont later in 2025
July 24, 2025 – amlodipine oral solution (Sdamlo)
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505(b)(2) NDA approval; amlodipine is also available as an oral tablet (Norvasc, generic), an oral 1 mg/mL solution (Norliqva) and a 1 mg/mL oral suspension (Katerzia)
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Calcium channel blocker
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Indicated alone or in combination with other antihypertensive and antianginal agents for the treatment of (1) hypertension, to lower blood pressure in adults and pediatric patients ≥ 6 years of age; lowering blood pressure reduces the risk of fatal and nonfatal CV events, primarily strokes and MIs and (2) coronary artery disease (CAD) in adults – chronic stable angina, vasospastic angina (Prinzmetal's or variant angina) and angiographically documented CAD in patients without HF or an ejection fraction < 40% to reduce the risk of hospitalization for angina and to reduce the risk of a coronary revascularization procedure
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Oral solution: 2.5 mg, 5 mg and 10 mg
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Recommended dosage is taken orally once daily with dosage dependent on patient’s age (pediatric or adult; small, fragile or elderly patients would start at the lower end of the dosage range)
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Product will be available from Brillian with launch timeframe TBD
July 29, 2025 – celecoxib oral suspension (Vyscoxa)
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505(b)(2) NDA approval; celecoxib is also available as oral capsules (Celebrex, generic) and an oral solution (Elyxyb); Elyxyb is indicated for the acute treatment of migraine with or without aura in adults
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Nonsteroidal anti-inflammatory drug (NSAID)
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Indicated in adults for (1) osteoarthritis, (2) rheumatoid arthritis and (3) ankylosing spondylitis and in pediatric patients ≥ 2 years of age for juvenile rheumatoid arthritis; not indicated for the management of acute pain or treatment of primary dysmenorrhea (celecoxib capsules have these indications)
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Limitations of use: must be administered on an empty stomach at least two hours before or one hour after food; taking with food leads to 50% higher plasma exposure levels; if not tolerated in the fasted state, discontinue use
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Oral suspension: 10 mg/mL
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Recommended dosage is taken orally once or twice daily with dosage dependent on indication; single doses > 200 mg (20 mL) are not recommended as these doses may result in celecoxib concentration higher than intended; a different celecoxib formulation should be used for patients who require a single dose higher than 200 mg (20 mL)
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Boxed warnings for risk of serious CV and GI events
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Product will be available from Carwin with the launch timeframe TBD
July 30, 2025 – methocarbamol oral suspension (Atmeksi)
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505(b)(2) NDA approval; methocarbamol is also available as a generic oral tablet and solution for injection (Robaxin, generics)
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Muscle relaxant
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Indicated as an adjunct to rest, physical therapy and other measures for the relief of discomfort associated with acute, painful musculoskeletal conditions in patients ≥ 16 years of age
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Oral suspension: 750 mg/5 mL
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Recommended initial dosage is taken orally four times daily and maintenance doses are taken every four hours or three times daily
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Product will be available from Metacel/Rosemont with launch timeframe TBD
Aug. 15, 2025 – cyclobenzaprine hydrochloride (HCl) sublingual tablets (Tonmya)
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505(b)(2) NDA approval; cyclobenzaprine is also available as a generic oral tablet and an extended release (ER) oral capsule (Amrix, generics); these products are not indicated for fibromyalgia
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Muscle relaxant
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Indicated for the treatment of fibromyalgia in adults
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Sublingual tablet: 2.8 mg
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Recommended initial dosage is one sublingual tablet once daily at bedtime for days one to 14, followed by an increase to two tablets once daily at bedtime beginning on day 15 and thereafter; ensure mouth is moist with sips of water prior to sublingual administration
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Product will be available from Tonix in Q4 2025
July 30, 2025 – bictegravir/emtricitabine/tenofovir alafenamide (Biktarvy)
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Gilead; three-drug combination of bictegravir, a HIV-1 integrase strand transfer inhibitor (INSTI), and emtricitabine (FTC) and tenofovir alafenamide (TAF), both HIV-1 nucleoside analog reverse transcriptase inhibitors (NRTIs)
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Expanded indication: as a complete regimen for the treatment of HIV-1 infection in adults and pediatric patients weighing ≥ 14 kg with an antiretroviral treatment history who are not virologically suppressed, with no known or suspected substitutions associated with resistance to the integrase strand inhibitor class, emtricitabine or tenofovir
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Administered orally as one tablet once daily with or without food; tablet strength is dependent on patient body weight
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Other indications are detailed in the product label
Aug. 5, 2025 – fremanezumab-vfrm (Ajovy)
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Teva; calcitonin gene-related peptide (CGRP) antagonist
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New indication: preventive treatment of episodic migraine in pediatric patients who are 6 to 17 years of age and who weigh ≥ 45 kg; not approved in pediatric patients weighing < 45 kg because of the lack of an appropriate strength presentation
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Administered as a SC monthly dosage; can be administered by (1) an HCP, (2) patients ≥ 13 years of age and/or (3) caregivers; pediatric patients 6 to 12 years of age require administration by an HCP or adult caregiver; proper training is required prior to self or caregiver administration
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Other indication: preventive treatment of migraine in adults
Aug. 15, 2025 – semaglutide (Wegovy)
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Novo Nordisk; glucagon-like peptide-1 (GLP-1) receptor agonist; Accelerated Approval
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New indication: in combination with a reduced calorie diet and increased physical activity for the treatment of noncirrhotic metabolic dysfunction-associated steatohepatitis (MASH), formerly known as nonalcoholic steatohepatitis (NASH), with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis) in adults; Accelerated Approval is based on improvement of MASH and fibrosis; therefore, continued approval for this use may require demonstration of benefit in confirmatory clinical trials
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Administered as a SC dosage once weekly on the same day each week, at any time of day, with or without meals; patients should receive training on proper injection technique prior to self-administration
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Other indications are detailed in the product label
First generic drug launches
July 22, 2025 – sertraline HCl oral capsule (no trade name)
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Zenara/Cipla launched a generic oral capsule (150 mg, 200 mg) to Almatica’s sertraline oral capsules
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Selective serotonin reuptake inhibitor (SSRI); indicated for the treatment of (1) major depressive disorder in adults and (2) obsessive-compulsive disorder in adults and pediatric patients ≥ 6 years of age
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Recommended dosage is one capsule once daily; treatment should not be initiated with sertraline HCl capsules; another sertraline HCl product should be used for the initial dosage, titration and dosages < 150 mg once daily
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Annual sales for sertraline capsules in 2024 were $33 million
July 23, 2025 – sacubitril/valsartan tablet (Entresto)
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Alkem/Ascend launched a generic oral tablet to Novartis’s Entresto
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Combination of sacubitril (a neprilisin inhibitor) and valsartan (an angiotensin II receptor blocker); indicated (1) to reduce the risk of CV death and hospitalization for HF in adults with chronic HF – benefits are most clearly evident in patients with left ventricular ejection fraction (LVEF) below normal and (2) for the treatment of symptomatic HF with systemic left ventricular systolic dysfunction in pediatric patients ≥ 1 year of age – sacubitril/valsartan reduces NT-proBNP and is expected to improve CV outcomes
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Recommended dosage is taken orally twice daily for adults and adjusted every two to four weeks to the target maintenance dose, as tolerated by the patient; dosage for pediatric patients is based on body weight taken orally twice daily and adjusted every two weeks, as tolerated by the patient; pediatric patients < 40 kg should use the oral suspension formulation
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Annual sales for Entresto tablets in 2024 were $6,558 million
COVID-19 coronavirus disease 2019
HER2 human epidermal growth factor receptor 2
HIV-1 human immunodeficiency virus type 1
mRNA messenger ribonucleic acid
NT-proBNP N-terminal pro b-type natriuretic peptide
RNA ribonucleic acid
Editor-In-Chief: Maryam Tabatabai, PharmD
Executive Editor: Anna Schreck Bird, PharmD
Deputy Editors: Nicole Kjesbo, PharmD, BCPS; Olivia Pane, PharmD, CDCES
The content in this publication is not a substitute for professional medical advice. For questions regarding any medical condition or if you need medical advice, please contact your health care provider.
All brand names are property of their respective owners.
All brand names are property of their respective owners.
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