FDA Grants New Indication to Omisirge for Severe Aplastic Anemia

On Dec. 5, 2025, the US Food and Drug Administration (FDA) approved Gamida Cell’s Omisirge^® (omidubicel-onlv) suspension for the treatment of severe aplastic anemia in patients six years and older following reduced intensity conditions and for whom a compatible donor is not available, marking the first cell therapy approved for this use. Severe aplastic anemia is a rare, life-threatening blood disorder in which the bone marrow fails to produce enough red blood cells, white blood cells and platelets, leading to anemia, increased risk of infections and bleeding complications. Currently, standard care relies on bone marrow or cord blood transplantation. Initially approved in 2023 to treat blood cancers, Omisirge uses banked umbilical cord blood that is altered using a form of vitamin B to increase and strengthen stem cells and natural killer (NK) cells in the transplant. Shipped frozen, specific protocols for thawing, diluting and giving it will limit Omisirge’s use to hospitals and cancer centers that have staff trained in its administration and management of any side effects that may occur. Omisirge is given as a single intravenous (IV) dose that contains a specific range of CD34+ cells after patients have been preconditioned with high doses of chemotherapy (chemo) that destroys cells in bone marrow (myeloablation). Approval was based on an ongoing, open-label, single-arm trial showing Omisirge provided early and sustained neutrophil engraftment in 12 of 14 patients with a median time to neutrophil recovery of 11 days. The most common adverse events with Omisirge were febrile neutropenia, viral and bacterial infections, hyperglycemia, immune thrombocytopenia and pneumonia, with autoimmune cytopenia occurring in 25% of patients. Labeling for Omisirge has boxed warnings concerning the possibilities that it may cause life-threatening infusion reactions, graft versus host disease (GvHD), engraftment syndrome or graft failure. Here is the prescribing information.

First Drug Approved Under New Expedited Pilot Program

On Dec. 9, 2025, the FDA approved USAntibiotics’ Augmentin XR^® (amoxicillin-clavulanate potassium) extended-release tablets, an oral antibiotic, for the treatment of community-acquired pneumonia and acute bacterial sinusitis in adults and pediatric patients. This marks the first approval under the Commissioner’s National Priority Voucher (CNPV) pilot program, an expedited review pathway that shortens the review timeline from the typical 10–12 months to just two months. This is a reintroduction of the extended-release brand that was previously discontinued by the manufacturer in 2011. The FDA stated that approval is intended to strengthen domestic antibiotic manufacturing at USAntibiotics’ Bristol, Tennessee facility, reducing reliance on fragile global supply chains and mitigating shortages. The recommended dose is one tablet every 12 hours for seven to 10 days, with standard precautions for beta-lactam antibiotics. This is the first approval of 15 CNPV vouchers the FDA has granted.

Blujepa Gets New Indication as First Oral Gonorrhea Treatment

The FDA on Dec. 11, 2025, approved a new indication for GSK’s Blujepa^® (gepotidacin), authorizing it as the first oral treatment for uncomplicated urogenital gonorrhea in adults and adolescents 12 years and older who weigh at least 45 kilograms and have few or no alternative options. The approval introduces the first new antibiotic class for gonorrhea in more than 30 years, providing an oral alternative to current injectable therapies. Health agencies, including the World Health Organization (WHO) and the Centers for Disease Control and Prevention (CDC), classify Neisseria gonorrhoeae as a priority drug-resistant pathogen, highlighting the need for new treatments. Approval is based on phase III data demonstrating non-inferiority to standard therapy (ceftriaxone plus azithromycin) with a 92.6% microbiological success rate. Blujepa was initially approved in early 2025 for uncomplicated urinary tract infections (uUTI). The recommended regimen for gonorrhea is two oral doses of 3,000mg (four 750mg tablets), administered approximately 10 to 12 hours apart. Full prescribing information, including warnings on QTc prolongation and drug interactions, is available here.

New Formulation of Orladeyo Approved for Children

The FDA has approved a new oral pellet formulation of BioCryst Pharmaceuticals’ Orladeyo^® (berotralstat) on Dec. 12, 2025, for prophylactic treatment of hereditary angioedema (HAE) in children aged two years to under 12 years. This is the first oral prophylactic therapy for this age group offering a sprinkle-like oral pellet formulation that allows for administration to be swallowed directly with water or milk, or sprinkled over soft, non-acidic food. Orladeyo, a plasma kallikrein inhibitor, was initially approved in 2020 for patients 12 years and older as a once-daily capsule. HAE often begins in childhood, and about 40% of affected children experience their first attack by five years of age. Approval was supported by interim data from the APeX-P trial, which demonstrated early and sustained reductions in monthly attack rates, a consistent safety profile and no new safety signals. The most common adverse event was nasopharyngitis. Launch is not yet known. Full prescribing information is available here.

Uplizna Gets New Indication for Myasthenia Gravis

On Dec. 11, 2025, the FDA approved Amgen’s Uplizna^® (inebilizumab-cdon) IV infusion for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive. Uplizna is the first CD19-targeted B-cell therapy for this indication, working by depleting CD19-positive B cells, addressing a biological root cause of gMG. Approval was based on results from the phase III Myasthenia Gravis Inebilizumab Trial (MINT), which demonstrated a 1.9-point improvement in Myasthenia Gravis Activities of Daily Living (MG-ADL) scores at 26 weeks versus placebo, with benefits sustained through 52 weeks in AChR-positive patients. The study also incorporated a steroid taper, with over 87% of patients reducing prednisone to 5mg or less per day by week 26. The recommended regimen includes two initial 300mg infusions two weeks apart, followed by a single 300mg infusion every six months. Premedication with corticosteroids, antihistamines and antipyretics is required to reduce infusion reactions. Previously approved for anti-aquaporin-4 (AQP4) positive neuromyelitis optica spectrum disorder (NMOSD) and immunoglobulin G4-related disease (IgG4-RD), this is Uplizna’s third indication. Updated prescribing information is available here.

New Formulation for Daybue Approved The FDA has approved a new oral powder formulation of Acadia Pharmaceuticals’ Daybue^® Stix (trofinetide) on Dec. 12, 2025. Approved for the treatment of Rett syndrome in adult and pediatric patients two years of age and older, this new formulation offers an alternative administration for caregivers and patients. Initially approved in 2023, Daybue was the first drug indicated to treat the underlying cause of Rett syndrome for patients at least two years old as standard 450mL bottles oforal solution containing 200mg/mL. This formulation will be available in 5,000mg, 6,000mg, and 8,000mg packets allowing caregivers to mix the powder with a variety of water-based liquids, providing flexibility to adjust taste and volume.  Almost exclusively affecting girls, Rett syndrome is caused by random mutations on X chromosomes. It is a progressive neurodevelopmental disorder that has a wide range of symptoms impacting multiple organ systems. The recommended weight-based dose is administered twice daily after mixing with liquid. Common adverse events include diarrhea and vomiting; caregivers should monitor hydration and gastrointestinal symptoms. Daybue Stix is expected to launch in the first quarter of 2026, and the current oral solution formulation will remain available. Here is the prescribing information.