FDA news in pediatrics: May 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 May 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 May, 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. INF108 to prevent necrotizing enterocolitis receives FDA designations

On May 7, 2025, Infinant Health announced that INF108, its investigational drug for necrotizing enterocolitis (NEC) prevention in preterm infants, received orphan drug and rare pediatric disease designations from the FDA.

These designations aim to support the development of INF108 for NEC, a life-threatening intestinal condition that affects 7% of very low birth weight infants in NICUs. The company plans to initiate clinical trials to evaluate INF108 in preterm infants.

2. FDA clears EN-374 therapy application for X-linked chronic granulomatous disease

On May 12, 2025, the FDA cleared an investigational new drug (IND) application for Ensoma’s EN-374, a first-in-class in vivo gene therapy for X-linked chronic granulomatous disease (X-CGD).

EN-374 uses virus-like particles to deliver a CYBB transgene directly to hematopoietic stem cells, aiming to restore immune function in X-CGD patients. The therapy, which received orphan drug and rare pediatric disease designations, will enter a Phase 1/2 trial in late 2025.

3. FDA approves pediatric indication for Optison ultrasound enhancing agent

On May 13, 2025, the FDA approved GE HealthCare’s Optison (Perflutren Protein-Type A Microspheres) for pediatric use to enhance echocardiogram clarity.
Already approved for adults, Optison now helps improve visualization of left ventricular structures in children with suboptimal echocardiograms, aiding cardiac diagnosis.

4. FDA starts review of nutrients in infant formula

On May 13, 2025, the FDA and HHS issued a request for information to begin a comprehensive review of infant formula nutrients under "Operation Stork Speed."
This marks the first full review since 1988 and aims to update nutrient standards, enhance transparency, and improve formula safety and effectiveness for U.S. infants.

5. FDA seeks to remove ingestible fluoride products for children

On May 13, 2025, the FDA and HHS announced plans to remove pediatric ingestible fluoride prescription drugs from the market due to safety concerns.
Citing links to gut microbiome disruption and thyroid disorders, the FDA will review evidence and seek public comment by October 31, 2025, before taking action.

6. FDA accepts NDA for berotralstat in HAE patients aged 2 to 11 years

On May 14, 2025, the FDA accepted a new drug application for berotralstat (ORLADEYO) for the treatment of hereditary angioedema in children aged 2 to 11, with priority review.

If approved, berotralstat would become the first targeted oral prophylactic therapy for pediatric HAE patients, with a decision expected by September 12, 2025.

7. FDA approves NVX-CoV2705 COVID-19 vaccine

On May 19, 2025, the FDA approved Novavax’s NVX-CoV2705 (Nuvaxovid), the only protein-based, non-mRNA COVID-19 vaccine in the United States, for at-risk patients.
The vaccine is indicated for individuals aged 12 to 64 with underlying conditions and all adults 65 and older, based on phase 3 trial data showing strong safety and efficacy.

8. FDA approves Jivi for patients aged 7 years and older with hemophilia A

On May 19, 2025, the FDA approved Jivi (antihemophilic factor [recombinant], PEGylated-aucl; Bayer) for children aged 7 to <12 with hemophilia A.
This expanded indication is based on clinical trial data showing Jivi’s safety and efficacy in reducing bleeding episodes in pediatric patients with severe disease.

9. FDA approves roflumilast foam 0.3% for scalp, body psoriasis

On May 22, 2025, the FDA approved roflumilast (Zoryve) topical foam 0.3% to treat plaque psoriasis of the scalp and body in patients aged 12 years and older.
In phase 2 and 3 trials, the foam demonstrated significant improvements in psoriasis symptoms, including rapid itch relief, with strong safety and tolerability data.

10. FDA approves MenQuadfi meningococcal vaccine for children 6 weeks or older

On May 23, 2025, the FDA expanded the approval of MenQuadfi (Sanofi) to include infants aged 6 weeks to 23 months for the prevention of meningococcal disease.

MenQuadfi is now the only quadrivalent MenACWY vaccine approved for use from 6 weeks of age, with data showing comparable safety and immune response to existing vaccines.

11. FDA approves hydrocortisone oral solution for children 5 years, older with adrenocortical insufficiency

On May 28, 2025, the FDA approved Eton Pharmaceuticals’ hydrocortisone oral solution (Khindivi) for pediatric patients aged 5+ with adrenocortical insufficiency.

Khindivi is the first FDA-approved hydrocortisone oral solution, offering precise, ready-to-use dosing tailored for children without the need for tablet splitting.