FDA news in pediatrics: August 2025

FDA news in pediatrics: August 2025 | Image credit: Contemporary Pediatrics

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 August 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 July 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: August 2025

1. FDA pulls clearance of Pfizer-BioNTech COVID-19 vaccine for children under 5

On August 27, 2025, the FDA authorized updated COVID-19 vaccines for the 2025–2026 season while significantly restricting use in children. Pfizer’s Comirnaty is now approved only for adults 65 and older and individuals aged 5–64 with underlying conditions, while Moderna’s Spikevax remains available for high-risk children 6 months to 11 years and Novavax’s Nuvaxovid for at-risk patients 12 and older.

The American Academy of Pediatrics (AAP) criticized the move, warning that reduced pediatric access creates dangerous vulnerabilities as respiratory virus season approaches. The AAP reaffirmed its recommendation for vaccination beginning at 6 months and emphasized that decisions should remain between parents and pediatricians.

2. FDA approves first RNA-targeted therapy for hereditary angioedema

On August 22, 2025, the FDA approved donidalorsen (Dawnzera; Ionis Pharmaceuticals) as the first RNA-targeted therapy for hereditary angioedema (HAE) prophylaxis in patients 12 years and older. Delivered via subcutaneous autoinjector every 4 or 8 weeks, donidalorsen significantly reduced attack rates in the phase 3 OASIS-HAE trial, with the Q4W regimen lowering attacks by 81% compared with placebo. Long-term extension data confirmed durable efficacy, and most patients reported preferring donidalorsen over prior prophylaxis.

The approval provides a new option for the estimated 7000 people in the US with HAE, a rare and potentially life-threatening condition. Experts emphasized that the therapy’s durable efficacy, quality-of-life improvements, and flexible dosing make it a meaningful advancement in long-term disease management.

3. FDA issues CRL for vatiquinone to treat Friedreich ataxia

On August 19, 2025, the FDA issued a complete response letter to PTC Therapeutics, declining approval of vatiquinone for Friedreich ataxia (FA) after determining that substantial evidence of efficacy was not demonstrated. Vatiquinone, a selective 15-lipoxygenase inhibitor, would have been the second approved therapy for FA following Biogen’s omaveloxolone.

The decision was based on data from the phase 3 MOVE-FA trial and long-term extension studies, which showed numerical but not statistically significant improvements in the primary end point. While nominal benefits were observed in measures of stability and fatigue, the FDA found these insufficient for approval. PTC stated it plans to meet with the agency to discuss next steps.

4. FDA approves first generic iron sucrose injection for iron deficiency anemia in CKD

ON August 12, 2025, the FDA approved Viatris Inc’s iron sucrose injection, USP, as the first generic version of Venofer for the treatment of iron deficiency anemia in adults and children ≥2 years with chronic kidney disease. The intravenous therapy will be available in multiple single-dose vial strengths and received a competitive generic therapy designation for select doses, granting 180 days of market exclusivity.

Viatris emphasized the approval as a milestone for patient access and its generics portfolio, noting the product was developed in-house. Iron sucrose carries risks of serious hypersensitivity reactions, hypotension, and iron overload, requiring patient monitoring. Common adverse effects include gastrointestinal symptoms, headache, dizziness, and injection site reactions, with safety profiles differing slightly between adults and pediatric patients.

5. FDA approves brensocatib to treat adolescents with non–cystic fibrosis bronchiectasis

On August 12, 2025, the FDA approved Insmed’s brensocatib (Brinsupri) as the first treatment for non–cystic fibrosis bronchiectasis (NCFB) in patients aged 12 years and older. The oral DPP1 inhibitor directly targets neutrophilic inflammation, a driver of bronchiectasis exacerbations, and was evaluated in the phase 3 ASPEN trial of more than 1700 patients.

Brensocatib significantly reduced annualized pulmonary exacerbation rates compared with placebo and prolonged the time to first exacerbation. Both 10-mg and 25-mg doses improved exacerbation-related outcomes, with the higher dose also slowing lung function decline. Experts described the approval as a paradigm shift, offering the first FDA-approved therapy for the estimated 500,000 people in the US living with NCFB.

6. FDA accepts priority review for gepotidacin as oral treatment for gonorrhea

On August 12, 2025, the FDA accepted GSK’s supplemental New Drug Application for gepotidacin (Blujepa) as an oral treatment for uncomplicated urogenital gonorrhea in patients ≥12 years weighing at least 45 kg, granting priority review with a target action date of December 11, 2025. Gepotidacin, already approved in March 2025 for uncomplicated urinary tract infections, would be the first oral therapy available for gonorrhea in the US if approved.

The submission is supported by phase 3 EAGLE-1 trial results, which showed gepotidacin was non-inferior to ceftriaxone plus azithromycin, achieving microbiological success rates above 90%. Safety findings were consistent with prior studies, with no severe treatment-related adverse events. An oral option could reduce dependence on injectable regimens, improving accessibility and adherence in gonorrhea management.

7. FDA approves tocilizumab-anoh for CRS in patients 2 years and older

On August 6, 2025, the FDA approved Celltrion’s tocilizumab-anoh (Avtozma) IV for cytokine release syndrome (CRS) in patients aged 2 years and older, bringing the biosimilar into full indication alignment with reference tocilizumab (Actemra). Tocilizumab-anoh, an interleukin-6 receptor antagonist, already carries approvals for polyarticular and systemic juvenile idiopathic arthritis, COVID-19, giant cell arteritis, and rheumatoid arthritis.

The IV formulation is expected to launch in the US on August 31, 2025, under a settlement agreement with Genentech. Celltrion stated that the approval expands access to high-quality biologics for CRS, a condition caused by excessive cytokine release that can lead to severe systemic complications.

8. FDA approves fremanezumab-vfrm for migraine prevention in children aged 6 years, older

On August 5, 2025, the FDA approved fremanezumab-vfrm (Ajovy; Teva Pharmaceuticals) for the preventive treatment of episodic migraine in children aged 6 years and older weighing at least 45 kg, making it the first calcitonin gene-related peptide (CGRP) therapy approved for pediatric migraine prevention. Previously indicated for adults, the drug is administered as a 225-mg monthly subcutaneous injection.

The approval was supported by phase 3 SPACE trial data, which showed significant reductions in monthly migraine days and a tolerability profile consistent with adult studies. Migraine affects nearly 8% of children, with higher prevalence in adolescents, and fremanezumab-vfrm now represents the only CGRP-targeted preventive option available across both pediatric and adult populations in the United States.