FDA news in pediatrics: April 2025 | Image Credit: © Araki Illustrations - © Araki Illustrations - stock.adobe.com.

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

Take a look at our detailed coverage of FDA-related news from April, 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: April 2025

1. FDA accepts sBLA to expand fremanezumab-vfrm indication for episodic migraines in children

On April 7, 2025, the FDA accepted a supplemental biologics license (sBLA) application for fremanezumab-vfrm (AJOVY; Teva Pharmaceuticals), to include children and adolescent patients aged 6 to 17 years with episodic migraine, who weigh 99 lbs (45 kgs) or more.

If approved, fremanezumab-vfrm would be the first calcitonin gene-related peptide (CGRP) antagonist for migraine prevention in adults and episodic migraine prevention in pediatric patients. Currently, fremanezumab-vfrm is the only anti-CGRP treatment for migraine prevention in adults in the United States that is available in both quarterly and monthly dosing options, according to an announcement from Teva.

2. FDA approves nivolumab with ipilimumab for MSI-H, dMMR colorectal cancer

On April 8, 2025, the FDA approved the combination of nivolumab (Opdivo; Bristol Myers Squibb) and ipilimumab (Yervoy; Bristol Myers Squibb) for use in adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC).

According to the announcement from the federal agency, it converted the previous accelerated approval for single-agent nivolumab to a regular approval for patients 12 and older with MSI-H or dMMR metastatic CRC that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.

3. FDA approves maralixibat tablet formulation for ALGS and PFIC

On April 8, 2025, the FDA approved the combination of nivolumab (Opdivo; Bristol Myers Squibb) and ipilimumab (Yervoy; Bristol Myers Squibb) for use in adult and pediatric patients 12 years and older with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (CRC).

According to the announcement from the federal agency, it converted the previous accelerated approval for single-agent nivolumab to a regular approval for patients 12 and older with MSI-H or dMMR metastatic CRC that has progressed following treatment with fluoropyrimidine, oxaliplatin, and irinotecan.

4. FDA expands diazepam nasal spray for seizure clusters to ages 2–5

On April 16, 2025, the FDA approved an expanded indication for diazepam nasal spray (Valtoco; Neurelis) for the short-term treatment of seizure clusters to include patients aged 2 years and older. The decision from the federal agency expands on the prior indication of immediate-use in patients aged 6 years and older.

According to a press release from Neurelis, Inc., treatment is intended for seizure clusters that are different from a person's normal seizure pattern. The proprietary formulation enables noninvasive intranasal delivery of diazepam. The intranasal formulation was previously recognized by the FDA as "clinically superior to the rectal gel formulation of diazepam, resulting in its orphan drug exclusivity designation," stated Neurelis.

5. ACIP votes in favor of recently FDA-approved vaccines for meningococcal disease, chikungunya

On April 17, the CDC's Advisory Committee on Immunization Practices (ACIP) voted to recommend the use of GSK's Meningococcal Groups A, B, C, W, and Y Vaccine (Penmenvy) be included in the adolescent meningococcal vaccination schedule. In addition, the ACIP voted to recommend use of Bavarian Nordic's chikungunya vaccine, recombinant (Vimkunya), in individuals aged 12 years and older who will travel to a country or territory where there is a chikungunya outbreak.

According to the CDC, recommendations made by the ACIP are pending and forwarded to the director of the CDC for potential final approval. If recommendations are adopted, they are then official CDC policy and are published in the agency's Morbidity and Mortality Weekly Report.

6. FDA approves dupilumab for chronic spontaneous urticaria in ages 12 and up

On April 18, 2025, the FDA approved a resubmitted supplemental biologics license application (sBLA) for dupilumab (Dupixent; Regeneron and Sanofi) to treat chronic spontaneous urticaria (CSU) in adults and pediatric patients aged 12 years and older, according to a press release from Regeneron. With the federal agency's decision, CSU is now the 7th disease with underlying type 2 inflammation in which dupilumab is approved.

The interleukin (IL)-4 and IL-13 inhibitor for this CSU indication is designed to treat those whose disease is not adequately controlled with H1 antihistamine treatment, and is the first targeted therapy for CSU in over a decade.

7. FDA issues CRL for aflibercept 8 mg extended dosing for all indications

On April 18, the FDA issued a complete response letter (CRL) to Regeneron Pharmaceuticals for a supplemental new drug application to add extended dosing intervals—up to every 24 weeks—to the label for EYLEA HD (aflibercept) Injection 8 mg, across all approved indications, according to a press release from the Pharmaceutical company.

Regeneron stated that the FDA’s response did not cite any concerns with the safety or efficacy of aflibercept at currently approved doses or intervals. However, the agency did not agree with the proposed addition of dosing intervals beyond 16 weeks, which is the longest interval presently indicated on the label. The company stated it is evaluating the federal agency's decision and "will determine a path forward in due course."

8. FDA announces measures to phase out petroleum-based food dyes

On April 22, the FDA and the US Department of Health and Human Services announced several new measures to phase out all petroleum-based synthetic dyes from the United States' food supply.

The FDA stated, in partnership with the NIH Regulatory Science and Research Program, will enhance nutrition and food-related research to inform regulatory decisions better.

9. FDA approves pz-cel for recessive dystrophic epidermolysis bullosa

On April 25, 2025, the FDA approved prademagene zamikeracel (Zevaskyn; Abeona Therapeutics) (pz-cel), as the first and only autologous, gene-modified cell therapy for adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), according to a press release from the company.

Pz-cel is now the first FDA-approved product designed to treat chronic RDEB wounds with a single application. RDEB is a rare and debilitating genetic skin disorder caused by mutations in the COL7A1 gene, leading to fragile skin that blisters and wounds easily.

10. FDA approves nipocalimab-aahu to treat generalized myasthenia gravis

On April 30, 2025, the FDA approved nipocalimab-aahu (Imaavy; Johnson & Johnson), a human FcRn-blocking monoclonal antibody, for the treatment of generalized myasthenia gravis (gMG) in adult and pediatric patients aged 12 years and older who are anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) antibody positive, according to a press release from the company.

The approval follows Priority Review designation and introduces a new treatment option in the FcRn inhibitor class. Nipocalimab-aahu is designed to selectively reduce immunoglobulin G (IgG) autoantibodies—without affecting other components of the immune system—offering targeted disease control in a broad patient population.

11. Elamipretide to treat Barth syndrome sees FDA target action delay

On April 29, Stealth BioTherapeutics announced that the FDA did not meet the April 29, 2025, Prescription Drug User Fee Action (PDUFA) date for elamipretide's new drug application (NDA) to treat Barth syndrome.

According to a press release from Stealth, the NDA was submitted to the FDA on January 29, 2024, with an initial PDUFA date of January 29, 2025. Following a new analyses request from the FDA in late November and early December of 2024, a delay in the target action date was announced, leading to a new PDUFA date of April 29, 2025.