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

As you continue to provide the best care possible for your patients, staying up to date on the quick-hitting headlines and larger key developments from the FDA can be challenging.

In this monthly recap, we provide our top FDA-related news items from the month, all in one list so you can stay in the loop with the ever-changing pediatric health care landscape.

This month, we highlighted the approval of a new 5-in-1 meningococcal vaccine, the first rapid-actig insulin biosimilar product for diabetes treatment, and much more.

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

FDA updates in pediatric care: February 2025

1. FDA expands immobilized lipase cartridge clearance down to 1 year

On January 15, 2025, Alcresta Therapeutics announced the FDA’s expanded clearance of the immobilized lipase cartridge (Relizorb) for use in children as young as 1 year old. Based on a retrospective evaluation of real-world data, the decision confirms no additional safety concerns in this younger population. This clearance marks another milestone for enterally fed patients with fat malabsorption, broadening access to essential nutritional support.

2. FDA approves mirdametinib for patients 2 years, older with neurofibromatosis type 1

On February 11, 2025, SpringWorks Therapeutics announced the FDA approval of mirdametinib (Gomekli) for adults and children aged 2 years and older with neurofibromatosis type 1 (NF1) and symptomatic plexiform neurofibromas (PN) not amenable to complete resection. Based on data from the phase 2b ReNeu trial, mirdametinib demonstrated an overall response rate of 41% in adults and 52% in pediatric patients, with responses assessed via volumetric MRI analysis. The approval provides a new treatment option for NF1-associated PN, though it carries risks of left ventricular dysfunction and ocular toxicities.

3. FDA approves GSK's meningococcal ABCWY vaccine

On February 14, 2025, GSK announced the FDA approval of the meningococcal ABCWY vaccine (Penmenvy) for individuals aged 10 to 25 years. Based on data from a phase 3 trial, the vaccine met all primary endpoints, demonstrating immunogenicity and a safety profile consistent with existing meningococcal vaccines. This approval provides a 5-in-1 option to protect against the most common invasive meningococcal disease-causing serogroups, potentially improving vaccination coverage in adolescents and young adults.

4. FDA approves insulin-aspart-szjj as biosimilar for diabetes treatment

On February 14, 2025, Sanofi-Aventis announced the FDA approval of insulin-aspart-szjj (Merilog) as the first rapid-acting insulin biosimilar for glycemic control in adults and pediatric patients with diabetes. Based on its demonstrated similarity to insulin aspart (Novolog), the approval includes both pre-filled pens and multi-dose vials for mealtime blood sugar management. This marks the third insulin biosimilar approved by the FDA, expanding treatment options for diabetes patients.

5. FDA accepts new drug application for vatiquinone to treat Friedreich's ataxia

On February 19, 2025, PTC Therapeutics announced the FDA’s acceptance of a New Drug Application for vatiquinone to treat Friedreich’s ataxia (FA), with priority review and a PDUFA target action date of August 19, 2025. Based on data from the phase 3 MOVE-FA trial and long-term studies, vatiquinone demonstrated potential benefits in slowing disease progression and improving fatigue and neurological symptoms in pediatric and adult patients. If approved, vatiquinone would be the first FDA-approved therapy for FA, addressing a significant unmet medical need.

6. FDA approves Surgifort human milk-based fortifier for term infants after gastroschisis surgery

On February 25, 2025, Prolacta Bioscience announced the FDA approval of Surgifort, the first human milk-based nutritional fortifier for term infants recovering from gastroschisis surgery. Based on its formulation of pasteurized human milk-derived nutrients, Surgifort is designed to support optimal growth and recovery by providing essential protein and calories. This approval expands human milk-based nutrition options beyond premature infants to a fragile post-surgical population.