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10 FDA Headlines You Missed in December 2025
This FDA News Month in Review provides a round-up of regulatory decisions from December 2025.
Welcome back to our recap of this month's news and updates from the US Food and Drug Administration (FDA)!
December 2025 was a landmark month for FDA approvals across rare diseases, hematology, pulmonology, cardiology, and metabolic disorders. Key approvals included first-in-class cellular and gene therapies: omidubicel-onlv for severe aplastic anemia and etuvetidigene autotemcel for Wiskott-Aldrich syndrome, both offering transplant-based options for patients without matched donors. Pediatric care advanced with berotralstat oral granules for children 2–11 years with hereditary angioedema, and narsoplimab for TA-TMA in patients aged ≥2 years, while mitapivat (AQVESME) addressed anemia in alpha- and beta-thalassemia, spanning transfusion-dependent and non–transfusion-dependent populations.
Other notable approvals expanded options for chronic and progressive diseases: inebilizumab-cdon for generalized myasthenia gravis, depemokimab-ulaa for severe eosinophilic asthma, nerandomilast for progressive pulmonary fibrosis, and aficamten for symptomatic obstructive hypertrophic cardiomyopathy, the latter with a REMS program due to heart failure risk. Metabolic disease management was transformed with the FDA’s approval of the first oral GLP-1 therapy for obesity, semaglutide (Wegovy), demonstrating robust weight loss in adults. Here’s a concise overview of the key FDA decisions and updates from December 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 Highlights: December 2025
1. FDA Approves Omidubicel-onlv (Omisirge) As First Cellular Severe Aplastic Anemia Therapy
On December 8, 2025, the FDA approved omidubicel-onlv as the first hematopoietic stem cell transplant therapy for patients with severe aplastic anemia who lack a compatible donor. The approval of the nicotinamide-enhanced cord blood therapy was supported by clinical trial data showing rapid neutrophil recovery, high transfusion independence, and low rates of graft-versus-host disease.
2. FDA Approves Etuvetidigene Autotemcel (Waskyra) For Wiskott-Aldrich Syndrome
On December 9, 2025, the FDA approved etuvetidigene autotemcel as the first cell-based gene therapy for Wiskott-Aldrich syndrome, offering a new option for pediatric and adult patients without a suitable matched donor for transplantation. Supported by clinical studies and expanded access data, the therapy demonstrated sustained reductions in severe infections and bleeding events by correcting the underlying genetic defect using a patient’s own modified stem cells.
3. FDA Approves Oral Berotralstat for Children Aged 2 – 11 Years with HAE
On December 12, 2025, the FDA approved berotralstat oral granules for the prevention of hereditary angioedema attacks in children aged 2–11 years, expanding access to the first oral prophylactic therapy for this population. Supported by interim data from the APeX-P trial, the approval addresses a longstanding gap in pediatric HAE care by offering a less burdensome, once-daily option with sustained reductions in attack rates. The decision extends the role of oral prophylaxis to younger patients who previously relied largely on injectable or infusion-based therapies.
4. FDA Approves Inebilizumab-cdon (Uplizna) for Generalized Myasthenia Gravis
On December 11, 2025, the FDA approved inebilizumab-cdon for the treatment of adults with generalized myasthenia gravis (gMG) who are anti-AChR or anti-MuSK antibody positive, expanding treatment options with a targeted therapy. Supported by data from the phase 3 MINT trial, the CD19-targeting monoclonal antibody demonstrated durable improvements in disease activity with a twice-yearly maintenance dosing schedule following initial loading.
5. FDA Approves Depemokimab-ulaa (Exdensur) for Severe Asthma
On December 16, 2025, the FDA approved depemokimab-ulaa as an add-on maintenance therapy for adults and adolescents aged 12 years and older with severe eosinophilic asthma. Supported by the phase 3 SWIFT-1 and SWIFT-2 trials, the ultra-long-acting biologic significantly reduced asthma exacerbations with just 2 doses per year, offering a lower-burden option compared with existing biologics.
6. FDA Approves Nerandomilast (Jascayd) Tablets for Progressive Pulmonary Fibrosis
On December 19, 2025, Boehringer Ingelheim announced FDA approval of nerandomilast (Jascayd) tablets for the treatment of progressive pulmonary fibrosis (PPF) in adults. Based on results from the phase 3 FIBRONEER-ILD trial—the largest clinical trial program conducted in PPF to date—the preferential PDE4B inhibitor significantly reduced the rate of lung function decline, as measured by forced vital capacity, compared with placebo over 52 weeks and is now the first and only PDE4B inhibitor with immunomodulatory and antifibrotic activity approved for PPF.
7. FDA Approves Aficamten (Myqorzo) for Symptomatic Obstructive Hypertrophic Cardiomyopathy
On December 19, 2025, the FDA approved Cytokinetics’ aficamten (Myqorzo) for adults with symptomatic obstructive hypertrophic cardiomyopathy to improve functional capacity and symptoms. The decision was based on phase 3 SEQUOIA-HCM results showing clinically meaningful improvements across exercise capacity, symptoms, and cardiac hemodynamics. The approval includes a Boxed Warning for heart failure risk and requires use under a REMS program.
8. FDA Approves Semaglutide (Wegovy) Pill As First Oral GLP-1 for Weight Loss
On December 22, 2025, the FDA approved Novo Nordisk’s once-daily oral semaglutide (Wegovy), the first oral GLP-1 therapy for obesity in the US. The approval, based on the 64-week phase 3 OASIS 4 trial, showed adults taking semaglutide with diet and exercise lost an average of 17% of body weight versus 3% with placebo, with 76% achieving at least 5% weight loss. The approval adds to its existing indication of reducing major cardiovascular events in adults with overweight or obesity and established cardiovascular disease.
9. FDA Approves Narsoplimab (Yartemlea) As First TA-TMA Therapy
On December 24, 2025, the FDA approved Omeros Corporation’s narsoplimab-wuug (Yartemlea) for the treatment of hematopoietic stem cell transplant–associated thrombotic microangiopathy (TA-TMA) in adults and children ≥2 years, making it the first and only approved lectin pathway inhibitor. The approval was based on a single-arm study and expanded access program showing complete response rates of 61–68% and 100-day survival of 73–74% in high-risk patients, including those refractory to prior therapies.
10. FDA Approves Mitapivat (AQVESME) for Non-Transfusion-Dependent Alpha- and Beta-Thalassemia
On December X, 2025, the FDA approved Agios’ mitapivat (AQVESME) for the treatment of anemia in adults with alpha- or beta-thalassemia, covering both transfusion-dependent and non–transfusion-dependent patients. Approval was supported by the phase 3 ENERGIZE and ENERGIZE-T trials, which demonstrated significant improvements in hemoglobin response and reductions in transfusion burden, respectively, with a manageable safety profile.
