FDA News Recap: March 2025

Welcome back to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!

March 2025 was a landmark month for FDA approvals, with game-changing therapies and medical innovations hitting the market. From the first new stroke treatment in decades to biosimilars expanding patient access, the agency delivered approvals that will shape healthcare for years to come.

Breakthroughs in Crohn’s disease, ATTR-CM, and C3 glomerulopathy introduced new standards of care, while AI-powered diagnostics and novel drug formulations pushed the boundaries of precision medicine. Not every decision was an approval—delays and regulatory challenges also made headlines. Here’s a look at the most significant FDA developments from March 2025.

Want to hear from the experts? Look for our Related Content links underneath blurbs for insight into how this decision might influence practice moving forward.

FDA News Month in Review: March 2025

FDA Approves Tenecteplase (TNKase) for Acute Ischemic Stroke

On March 3, 2025, Genentech announced that the FDA approved tenecteplase (TNKase) for the treatment of acute ischemic stroke (AIS), marking the first new stroke therapy approval in nearly 30 years. Tenecteplase, a tissue plasminogen activator, is administered as a single 5-second intravenous bolus, offering a faster and simpler alternative to alteplase. The approval was based on the phase 3 AcT trial, which demonstrated noninferiority to alteplase in efficacy and safety.

FDA Approves Celltrion’s Denosumab Biosimilars for Bone Loss Indications

On March 3, 2025, Celltrion announced that the FDA approved denosumab-bmwo under the names Stoboclo and Osenbelt, a biosimilar to denosumab, for all approved reference indications, including osteoporosis and cancer-related bone loss. The approval was supported by phase 3 trial data demonstrating comparable efficacy, safety, and immunogenicity to the reference product. The biosimilars are expected to launch in the United States in June 2025.

FDA Approves First Generic of Rivaroxaban (Xarelto) for CAD, PAD On March 4, 2025, the FDA approved the first generic formulation of rivaroxaban (Xarelto) 2.5 mg tablets, developed by Taro Pharmaceuticals, for reducing cardiovascular risk in adults with coronary artery disease (CAD) and thrombotic risk in adults with peripheral artery disease (PAD). The approval was supported by data from the COMPASS and VOYAGER-PAD trials, which demonstrated the efficacy of low-dose rivaroxaban combined with aspirin in lowering major cardiovascular and thrombotic events. The FDA emphasized that expanding access to safe and effective generics remains a priority to improve patient care.

FDA Approves 1 mg neffy Nasal Spray for Pediatric Anaphylaxis On March 5, 2025, ARS Pharmaceuticals announced FDA approval of epinephrine nasal spray 1 mg (neffy) for treating type 1 allergic reactions, including anaphylaxis, in patients aged 4 years and older weighing 15 to less than 30 kg. The approval follows the August 2024 authorization of the 2 mg formulation for older children and adults. Supporting data included pharmacokinetic and pharmacodynamic studies demonstrating efficacy comparable to injectable epinephrine.

FDA Approves Revakinagene Taroretcel (ENCELTO) for MacTel

On March 6, 2025, Neurotech Pharmaceuticals announced FDA approval of revakinagene taroretcel-lwey (ENCELTO), the first and only treatment for Macular Telangiectasia type 2 (MacTel), a neurodegenerative retinal disease that causes progressive vision loss. Utilizing encapsulated cell therapy (ECT) technology, the implant delivers continuous therapeutic doses of ciliary neurotrophic factor (CNTF) to slow disease progression. Approval was based on positive phase 3 trial results demonstrating significant preservation of macular photoreceptors over 24 months, with availability expected in the U.S. by June 2025.

FDA Approves Furosemide Injection (Furoscix) for Edema in CKD

On March 6, 2025, scPharmaceuticals announced FDA approval of its supplemental New Drug Application (sNDA) for furosemide (Furoscix), expanding its indication to include the treatment of edema in patients with chronic kidney disease (CKD). The approval allows subcutaneous furosemide injection (80 mg/10 mL) to be used for fluid overload management in adults with chronic heart failure or CKD, including nephrotic syndrome. Expected to launch in April 2025, this expansion builds on prior approvals and supports at-home IV-equivalent diuresis, with ongoing development of an autoinjector for low-volume administration.

FDA Approves First Interchangeable Biosimilar for Omalizumab

On March 9, 2025, Celltrion announced FDA approval of omalizumab-igec (OMLYCLO) as the first interchangeable biosimilar to omalizumab (Xolair) and the first respiratory biosimilar for multiple allergic and respiratory conditions, including asthma and chronic spontaneous urticaria. Approval was based on a phase 3 trial demonstrating bioequivalence in chronic spontaneous urticaria, with similar efficacy, safety, and immunogenicity to Xolair. The designation may expand access and lower costs, particularly for patients with IgE-mediated food allergies, following omalizumab’s 2024 approval for reducing allergic reactions after accidental food exposure.

FDA Clears AI-Assisted CaRi-Plaque Image Analysis for CAD

On March 11, 2025, Caristo Diagnostics announced FDA 510(k) clearance for its AI-assisted CaRi-Plaque image analysis application, designed to assess coronary plaque and luminal stenosis from routine CCTA scans. The technology enables early detection of coronary artery disease by identifying plaque buildup and inflammation, potentially guiding earlier intervention and treatment. This approval expands the company's AI-powered cardiovascular imaging portfolio, following its CaRi-Heart platform, which has demonstrated predictive value for major adverse cardiovascular events.

FDA Approves Guselkumab (Tremfya) For Crohn Disease On March 20, 2025, Johnson & Johnson announced FDA approval of guselkumab (Tremfya) for moderately to severely active Crohn’s disease, marking its fourth indication. The approval was supported by data from multiple Phase 3 trials, including GRAVITI and GALAXI studies, which demonstrated superiority over ustekinumab (Stelara) in achieving clinical remission and endoscopic response. Guselkumab, the first IL-23 inhibitor with both subcutaneous and intravenous induction options, offers a flexible treatment approach for patients with Crohn disease.

FDA Approves Oral Iptacopan (Fabhalta) as First C3 Glomerulopathy Therapy On March 20, 2025, Novartis announced FDA approval of oral iptacopan (Fabhalta) for reducing proteinuria in adults with C3 glomerulopathy (C3G), marking the first approved treatment for this ultra-rare kidney disease. The approval was based on the Phase 3 APPEAR-C3G trial, which demonstrated a 35.1% reduction in 24-hour urine protein-to-creatinine ratio at six months, with sustained benefit at 12 months. This marks the second rare kidney disease indication for iptacopan, following its 2024 approval for primary IgA nephropathy.

FDA Approves Vutrisiran (AMVUTTRA) for ATTR-CM

On March 20, 2025, Alnylam announced FDA approval of vutrisiran (AMVUTTRA) for the treatment of adults with cardiomyopathy due to wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM). This makes vutrisiran the first approved RNAi therapeutic targeting cardiovascular mortality, hospitalizations, and heart failure visits in ATTR-CM. Approval is based on the phase 3 HELIOS-B trial, which showed a 28% reduction in all-cause mortality and cardiovascular events.

FDA Approves Gepotidacin (Blujepa) for Uncomplicated UTI

On March 25, 2025, the US FDA approved gepotidacin (Blujepa) for the treatment of uncomplicated urinary tract infections (uUTIs) in female adults and adolescents. This approval follows positive results from the Phase 3 EAGLE-2 and EAGLE-3 trials, where gepotidacin demonstrated non-inferiority or superiority to nitrofurantoin, the current standard of care. Gepotidacin, a first-in-class antibiotic, works by inhibiting bacterial DNA replication and is effective against most uropathogens, including drug-resistant strains.

FDA Issues CRL for Etripamil Nasal Spray (Cardamyst) in PSVT On March 28, 2025, the US FDA issued a Complete Response Letter (CRL) for Milestone Pharmaceuticals' etripamil nasal spray (Cardamyst) for the treatment of paroxysmal supraventricular tachycardia (PSVT). The CRL did not raise concerns regarding the drug's clinical safety or efficacy but highlighted two key Chemistry, Manufacturing, and Controls issues, including the need for additional information on nitrosamine impurities and a facility inspection. Milestone plans to request a Type A meeting to address these issues and move forward with a resubmission.

FDA Approves Diazoxide Choline XR for Hyperphagia in Prader-Willi Syndrome On March 26, 2025, the US FDA approved diazoxide choline extended-release tablets (Vykat XR) for the treatment of hyperphagia in adults and children aged 4 years and older with Prader-Willi syndrome (PWS). The approval, based on data from the phase 3 C602-RWP trial, marks the first FDA-approved therapy to address hyperphagia in PWS, demonstrating significant improvement in hyperphagia-related behaviors compared to placebo (P = .0022).

Fitusiran Approved for People With Hemophilia A or B, With and Without Inhibitors On March 28, 2025, the US FDA approved fitusiran (Qfitlia) for routine prophylaxis to reduce bleeding episodes in patients aged 12 years and older with hemophilia A or B, with or without inhibitors. The approval was based on data from the phase 3 ATLAS program, where fitusiran prophylaxis demonstrated a 70% reduction in annualized bleeding rate compared to bypassing agent prophylaxis (P = .0002). Sanofi’s therapy offers a less frequent dosing regimen, with some patients requiring as few as six doses per year.

Related Content: Adding Treatment Options to the Hemophilia Landscape With Fitusiran’s Approval, with Margaret Ragni, MD, MPH