Another month has come and gone, and with it, several FDA approvals and regulatory updates related to the ever-evolving landscape of pediatric health care.

In our December monthly recap, we list our top FDA-related news items in a quick, easy-to-read format so you can stay informed.

Take a look at our detailed coverage of FDA-related news from November and easily stay in touch with our digital newsletters that bring you practical information for today's pediatrician.

Click the title of each story below for our full coverage of that regulatory update.

FDA updates in pediatric care: December 2025

1. FDA expands indication for MED-EL cochlear implants to infants 7 months or older

On December 4, 2025, the U.S. Food and Drug Administration approved an expanded indication for MED-EL cochlear implants, authorizing their use in children as young as 7 months with bilateral sensorineural hearing loss, making it the earliest FDA-approved cochlear implant option for infants. The decision was supported by pediatric clinical trial data demonstrating high rates of safety and effectiveness in children who did not receive adequate benefit from hearing aids, reinforcing the importance of early auditory access during critical periods of speech and language development. The expanded labeling also broadens audiologic and speech indications for older children, potentially enabling earlier intervention for a wider pediatric population with significant hearing loss.

2. FDA approves omidubicel-onlv as first HSCT therapy to treat severe aplastic anemia

On December 8, 2025, the FDA approved omidubicel-onlv (Omisirge; Gamida Cell), marking the first hematopoietic stem cell transplant therapy authorized for patients with severe aplastic anemia who lack a compatible donor. The approval was supported by clinical data demonstrating rapid and sustained neutrophil engraftment following reduced-intensity conditioning, addressing key limitations of traditional umbilical cord blood transplantation, such as delayed hematopoietic recovery and infection risk. Interim data presented at the 2025 American Society of Hematology Annual Meeting further showed high survival rates, low rates of graft-vs-host disease, and rapid recovery, positioning omidubicel-only as a new treatment option for a population with significant unmet need.

3. FDA approves Waskyra as first gene therapy for Wiskott-Aldrich syndrome

On December 9, 2025, the FDA approved Waskyra (etuvetidigene autotemcel; Fondazione Telethon), marking the first cell-based gene therapy authorized in the United States for the treatment of Wiskott-Aldrich syndrome. The approval was supported by clinical trial and expanded access data demonstrating substantial and durable reductions in severe infections and bleeding events following treatment with autologous, genetically corrected hematopoietic stem cells. The decision introduces a first-in-class option for pediatric and adult patients with WAS who lack a suitable matched donor, addressing a major unmet need in this rare, life-threatening immunodeficiency.

4. FDA approves gepotidacin as oral treatment for urogenital gonorrhea

On December 11, 2025, the FDA approved a supplemental new drug application for gepotidacin (Blujepa; GSK) as an oral treatment option for uncomplicated urogenital gonorrhea in patients aged 12 years and older with limited or no alternative therapies. The approval was supported by phase 3 EAGLE-1 trial data demonstrating noninferior microbiological cure rates compared with injectable ceftriaxone plus azithromycin, with no treatment-related serious adverse events reported. As the first new class of antibiotics for gonorrhea in more than three decades, gepotidacin provides a noninjectable option amid rising antimicrobial resistance and ongoing challenges with access to standard injectable therapy.

5. FDA approves berotralstat for patients aged 2 to 11 years with hereditary angioedema

On December 12, 2025, the FDA approved BioCryst’s berotralstat (Orladeyo) oral pellets for prophylactic treatment of hereditary angioedema in pediatric patients aged 2 to 11 years, making it the first targeted oral preventive therapy available for children younger than 12 with HAE. The decision was supported by interim phase 3 APeX-P trial data showing a favorable safety profile and early, sustained reductions in monthly attack rates, addressing a significant unmet need in a rare, potentially life-threatening disease that often begins in early childhood.

6. FDA approves powder formulation of trofinetide for Rett syndrome

On December 12, 2025, the FDA as approved a new powder-for-oral-solution formulation of trofinetide (Daybue Stix; Acadia Pharmaceuticals) for the treatment of Rett syndrome in adult and pediatric patients aged 2 years and older. The dye- and preservative-free formulation was shown to be bioequivalent to the approved oral solution, supporting comparable efficacy and safety without the need for additional clinical trials, and offers greater flexibility in dose volume and administration. As the first and only FDA-approved therapy for Rett syndrome, the expanded formulation may help address feeding challenges and support more individualized care for patients and caregivers managing this rare neurodevelopmental disorder.

7. FDA approves Nuzolvence (zoliflodacin) for treatment of uncomplicated urogenital gonorrhea in 12 years and up

On December 12, 2025, the FDA approved Nuzolvence (zoliflodacin; Innoviva Specialty Therapeutics) as a single-dose oral treatment for uncomplicated urogenital gonorrhea in adults and pediatric patients aged 12 years and older who weigh at least 35 kg, representing one of the first new treatment options for this infection in nearly two decades. The approval was supported by multinational phase 3 trial data demonstrating noninferiority to standard injectable therapy, including activity against drug-resistant Neisseria gonorrhoeae. The decision introduces a noninjectable alternative at a time of rising antimicrobial resistance and limited therapeutic options, with potential to expand access to timely treatment in a range of clinical settings.

8. FDA approves depemokimab for severe asthma in patients 12 years or older

On December 16, 2025, the FDA approved depemokimab-ulaa (Exdensur; GSK) as an add-on maintenance treatment for severe asthma with an eosinophilic phenotype in adults and pediatric patients aged 12 years and older. The decision was supported by phase 3 SWIFT-1 and SWIFT-2 trial data showing significant reductions in annualized asthma exacerbations with twice-yearly dosing compared with placebo plus standard of care, along with fewer exacerbations requiring hospitalization or emergency department visits. As the first FDA-approved ultra–long-acting biologic for severe asthma, depemokimab-ulaa offers a lower-frequency dosing option that may improve treatment adherence and sustained exacerbation control.

9. Abbott receives FDA clearance for Amplatzer Piccolo Delivery System for PDA closure in infants

On December 18, 2025, the FDA granted clearance to Abbott for a new Amplatzer Piccolo Delivery System designed to facilitate patent ductus arteriosus (PDA) closure in premature infants weighing as little as 2 lb. The delivery system enables single-catheter implantation and more precise device positioning in very small patients, addressing procedural challenges unique to neonatal care. The clearance expands Abbott’s pediatric structural heart portfolio and supports less invasive PDA closure in a highly vulnerable infant population.

10. FDA approves fibrinogen, human-chmt for acute bleeding in congenital fibrinogen deficiency

On December 19, 2025, the FDA approved fibrinogen, human-chmt (Fesilty; Grifols) for the treatment of acute bleeding episodes in pediatric and adult patients with congenital fibrinogen deficiency, including hypo- and afibrinogenemia. The plasma-derived fibrinogen concentrate was supported by phase I/III clinical data demonstrating effective and predictable restoration of fibrinogen levels, addressing limitations of fresh frozen plasma and cryoprecipitate in time-sensitive bleeding emergencies. The approval introduces a new targeted option for managing acute bleeding in this rare inherited disorder, with US commercial availability expected in the first half of 2026.

11. FDA approves ferric maltol for iron deficiency in patients 10 years and up

On December 22, 2025, the FDA approved an expanded indication for ferric maltol (ACCRUFeR; Shield Therapeutics), extending use of the oral iron therapy to pediatric patients aged 10 years and older with iron deficiency following a priority review. The decision was supported by phase 3 pediatric and adolescent data demonstrating clinically meaningful hemoglobin increases with a favorable tolerability profile, addressing adherence challenges seen with traditional iron salts. The approval broadens access beyond adults and positions ferric maltol as an FDA-approved oral option for adolescents with iron deficiency, with the sponsor planning further submissions to extend use to younger children.

12. FDA approves ferric maltol for iron deficiency in patients 10 years and up

On December 23, 2025, the FDA approved an expanded indication for furosemide injection (FUROSCIX; MannKind) on-body infusor to include pediatric patients who weigh 43 kg or more. The decision extends the subcutaneous therapy’s use beyond adults with chronic heart failure or chronic kidney disease, fulfilling postmarketing requirements under the Pediatric Research Equity Act. The approval provides a nonhospital option for managing edema, fluid overload, or hypervolemia in eligible pediatric patients, offering clinicians greater flexibility in treating congestion while potentially reducing the need for inpatient care.