7 FDA Headlines You Missed in September 2025

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

September 2025 saw the FDA advance treatment options across a range of rare and chronic conditions, with approvals spanning hematology, dermatology, cardiology, gastroenterology, lipid disorders, and endocrinology. Early in the month, recombinant von Willebrand factor (Vonvendi) gained expanded approval for prophylaxis in adults with all VWD subtypes and on-demand and perioperative use in children, making it the first non-plasma-derived VWF available for pediatric patients. Krystal Biotech’s beremagene geperpavec-svdt (Vyjuvek) also received a label update, enabling use from birth and at-home caregiver application in dystrophic epidermolysis bullosa.

The FDA further broadened access to innovative therapies with bumetanide nasal spray for edema, ruxolitinib cream for pediatric atopic dermatitis, and a subcutaneous induction option for guselkumab in ulcerative colitis. Pediatric expansions included evinacumab for children as young as one with homozygous familial hypercholesterolemia. Finally, paltusotine became the first once-daily oral treatment for adults with acromegaly, offering an alternative to injectable therapies. Here’s a concise overview of the key FDA decisions and updates from September 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 Recap: September 2025

1. FDA Expands Approval for Recombinant VWF, Includes Pediatric Patients

On September 5, 2025, the FDA expanded approval of recombinant von Willebrand factor (Vonvendi) to include prophylaxis in adults with all types of von Willebrand disease (VWD) and on-demand and perioperative use in children, making it the first non-plasma-derived VWF product available for pediatric patients. Previously limited to adults, recombinant VWF now establishes prophylaxis as a standard of care across VWD subtypes, supported by phase 3 trial and real-world data. With its long half-life and single-infusion efficacy in most bleeds, the expanded approval significantly broadens treatment access for patients with this rare bleeding disorder.

2. FDA Approves Beremagene Geperpavec-svdt (Vyjuvek) Label Update in DEB
On September 15, 2025, the FDA approved a label update for Krystal Biotech’s beremagene geperpavec-svdt (Vyjuvek), expanding use to dystrophic epidermolysis bullosa (DEB) patients from birth and allowing at-home caregiver application. The update also permits greater flexibility in wound dressing changes, easing treatment integration into daily care routines. Backed by real-world evidence and extension study data, the decision reinforces beremagene geperpavec's long-term safety and efficacy while enhancing convenience and quality of life for patients with broader access and flexibility across all ages and care settings.

3. FDA Approves Bumetanide Nasal Spray for Edema with Congestive Heart Failure

On September 15, 2025, the FDA approved Corstasis Therapeutics’ bumetanide nasal spray (Enbumyst) for edema associated with congestive heart failure, liver disease, and chronic kidney disease. Developed to offer an alternative to oral and intravenous loop diuretics, the intranasal formulation demonstrated bioequivalence to oral bumetanide in the RSQ-777-02 trial, with fewer adverse events and more consistent absorption. By enabling earlier outpatient intervention and reducing reliance on hospital-based IV therapy, bumetanide nasal spray provides a novel approach to managing fluid overload in patients at high risk of hospitalization and readmission.

Watch now: Bumetanide Nasal Spray and the Future of Edema With Josephine Harrington, MD, and Nihar Desai, MD

4. FDA Approves Ruxolitinib Cream for Pediatric Patients with Atopic Dermatitis

On September 18, 2025, the FDA approved ruxolitinib cream (Opzelura) for children aged 2–11 years with moderate-to-severe atopic dermatitis not adequately controlled with topical prescription therapies. The decision was supported by the phase 3 TRuE-AD3 trial, which showed sustained disease control and a favorable safety profile over 52 weeks, with most patients spending nearly half the treatment period off therapy due to lesion clearance. This expands access to a non-steroidal JAK1/2 inhibitor option for younger children, building on prior approvals in older pediatric patients.

Watch now: Reacting to FDA Approval of Ruxolitinib for Pediatric Atopic Dermatitis, with Amy Paller, MD

5. FDA Approves Guselkumab (Tremfya) Subcutaneous Induction Regimen for Ulcerative Colitis

On September 19, 2025, the FDA approved a subcutaneous induction regimen of guselkumab (Tremfya) for adults with moderately to severely active ulcerative colitis, making it the first IL-23 inhibitor with both subcutaneous and intravenous induction options for ulcerative colitis and Crohn’s disease. The approval was supported by the phase 3 ASTRO trial, which demonstrated significant improvements in clinical remission and endoscopic outcomes compared to placebo. Guselkumab separated from placebo as early as two weeks and maintained benefit through week 24, providing patients a more flexible initiation option alongside the established intravenous regimen.

Watch now: Guselkumab Subcutaneous Induction Advances Ulcerative Colitis Care, With David Rubin, MD

6. FDA Approves Evinacumab (Evkeeza) for HoFH in Children As Young As 1 Year Old

On September 26, 2025, the FDA expanded approval of evinacumab-dgnb (Evkeeza) to include children aged 1 to <5 years with homozygous familial hypercholesterolemia, making it the first ANGPTL3 antibody available for this age group. Already approved for older pediatric and adult patients, evinacumab lowers LDL-C by blocking ANGPTL3, a key regulator of lipid metabolism. The decision was supported by safety and pharmacokinetic data from compassionate use programs in young children, with no new safety concerns identified. This extension provides the earliest treatment option yet for children with this life-threatening genetic condition.

7. FDA Approves Paltusotine (Palsonify) As First Once-Daily, Oral Acromegaly Treatment

On September 25, 2025, the FDA approved Crinetics Pharmaceuticals’ paltusotine (Palsonify) as the first once-daily oral treatment for adults with acromegaly who are not candidates for surgery or have had inadequate surgical response. The approval was supported by the phase 3 PATHFNDR-1 and PATHFNDR-2 trials, where paltusotine achieved significant and durable biochemical control, with 83% of patients in PATHFNDR-1 maintaining normal IGF-1 levels compared to 4% on placebo. As a selective SST2 agonist, paltusotine offers a convenient alternative to injectable therapies.