Revuforj Gets New Indication for NPM1-Mutated AML

On Oct. 24, 2025, the FDA approved Syndax Pharmaceuticals’ Revuforj^® (revumenib), an oral menin inhibitor, for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML) in adult and pediatric patients aged one year and older with a susceptible nucleophosmin 1 (NPM1) mutation in which no satisfactory alternative treatment options exist. Revuforj is the first therapy approved specifically for R/R AML with NPM1 mutations, expanding its previous indication for R/R acute leukemia with KMT2A translocation, which was also the first therapy for that subtype. NPM1 mutations, found in about 30% of adult AML cases, are associated with aggressive disease and poor outcomes. Standard treatment for most leukemias includes chemotherapy, often followed by stem cell transplantation. Approval was based on phase II data demonstrating a 23% complete remission (CR) plus CR with partial hematological recovery (CRh) rate, with a median time to response of 2.8 months and median duration of 4.5 months. Notably, 17% of transfusion-dependent patients achieved transfusion independence. Revuforj carries a boxed warning for potentially fatal differentiation syndrome, along with warnings for QTc interval prolongation and embryo-fetal toxicity. The recommended dose is based on the patient’s weight and if they are concomitantly taking a strong CYP3A4 inhibitor. It is administered orally twice a day, either fasting or with a low-fat meal. Revuforj is now included in the National Comprehensive Cancer Network (NCCN) Guidelines as a Category 2A recommendation for NPM1-mutated AML. Here is the updated prescribing information.

FDA Expands Winrevair’s Indication to Reduce PAH-Related Mortality

The FDA approved an updated indication for Merck’s Winrevair^™ (sotatercept-csrk) 45mg and 60mg, on Oct. 27, 2025, for the treatment of adults with pulmonary arterial hypertension (PAH), World Health Organization (WHO) Group 1 to improve exercise capacity, WHO functional class, and now, to reduce the risk of clinical worsening events, including hospitalization for PAH, lung transplantation and death. This approval expanded the indication to include components of the clinical worsening events: hospitalization for PAH, lung transplantation and death. The expanded label is supported by the phase III ZENITH trial, which showed a 76% reduction in major morbidity and mortality outcomes in patients who have WHO functional class III or IV PAH treated with Winrevair compared to placebo. The trial was stopped early due to overwhelming efficacy, and patients were offered open-label follow-up. Winrevair is a fusion protein based on the activin receptor type IIA (ActRIIA), which is the natural high affinity receptor for activin and other proteins in the Transforming Growth Factor-beta (TGF-beta) superfamily. By combining parts of human ActRIIA and immunoglobulin G1 (IgG1), it blocks the activity of activins and growth differentiation factors that contribute to PAH. The recommended starting dose is 0.3mg/kg by subcutaneous (SC) injection every three weeks, which can be further increased to the target dose of 0.7mg/kg every three weeks, labs permitting. The patient’s hemoglobin (Hgb) and platelet levels should be evaluated before receiving at least the first five doses, and then periodically thereafter to determine if dose adjustments are needed. The updated prescribing information is here.

Omvoh Approved as a Single-Injection Maintenance Injection for Ulcerative Colitis

On Oct. 27, 2025, the FDA approved Omvoh^® (mirikizumab-mrkz – Eli Lilly) 200mg/2mL SC injection as a once-monthly maintenance regimen for adults with moderately to severely active ulcerative colitis (UC). This new single-injection formulation replaces the previous regimen that consisted of two consecutive 100mg/mL injections. Approval was based on a phase I pharmacokinetic study demonstrating bioequivalence between the single and dual injection formats. Omvoh induction begins with three 300mg intravenous (IV) infusions every four weeks, followed by monthly subcutaneous maintenance. It is also approved for Crohn’s disease. Common side effects include upper respiratory infections, injection site reactions and joint pain. Serious risks include allergic reactions, infections and liver issues. The new maintenance dose, Omvoh 200mg/mL citrate-free, will be available in the US via prefilled pen or prefilled syringe in early 2026. Here is the prescribing information.

FDA Grants Interchangeability to Celltrion’s Denosumab Biosimilars

The FDA designated Celltrion’s Stoboclo^® (denosumab-bmwo) and Osenvelt^® (denosumab-bmwo) as interchangeable biosimilars to Prolia^® and Xgeva^® respectively, for all approved indications on Oct. 29, 2025. In states that allow biosimilar substitution, Stoboclo and Osenvelt can now be dispensed instead of Prolia and Xgeva respectively, without contacting the prescriber for permission to substitute. Stoboclo (60mg/mL injection) is indicated for osteoporosis-related conditions, while Osenvelt (120mg/1.7mL injection) targets cancer-related bone loss and hypercalcemia of malignancy. Interchangeability designations were supported by phase III trial data evaluating efficacy, pharmacodynamics, pharmacokinetics, safety and immunogenicity to its reference product. Under new FDA draft guidance, biosimilar applicants may now seek interchangeability using existing Biologics License Application (BLA) data, rather than conducting additional switching studies previously required.

Recall

Additional Lots of Gamunex-C Recalled       

On Oct. 28, 2025, Grifols Therapeutics LLC initiated a voluntary withdrawal of four lots of Gamunex-C^® (immune globulin injection [human], 10%) after identifying an increased rate of allergic and hypersensitivity reactions associated with these vials. It follows a series of earlier withdrawals for the same safety concern on Feb. 19, March 21, Aug. 8 and Sept. 30, 2025, underscoring ongoing vigilance around hypersensitivity risks. Gamunex-C is indicated for the treatment of primary humoral immunodeficiency (PI) in patients aged two years and older, idiopathic thrombocytopenic purpura (ITP) in both pediatric and adult populations and chronic inflammatory demyelinating polyneuropathy (CIDP) in adults. While hypersensitivity and anaphylactic reactions are already noted in the product’s prescribing information under warnings and precautions, the withdrawn lots may carry an elevated risk for these adverse events. The October withdrawal was conducted with FDA knowledge and requires action down to the consumer level. For full prescribing details, see here.