FDA news in pediatrics: January 2025

FDA news in pediatrics: January 2025 Image Credit: Adobe Stock

Navigating the ever-evolving landscape of pediatric care means staying ahead of clinical developments, regulatory shifts, and therapeutic breakthroughs—each with the potential to reshape how we support children’s health. In the January 2025 FDA update from Contemporary Pediatrics, we highlight decisions that reflect both the complexity and promise of modern pediatrics, including new drug submissions, a pivotal ban on red No. 3 in food and ingested drugs, and an expanded clearance for a familiar face.

Dive into the details in our monthly FDA recap, and stay connected with Contemporary Pediatrics through our newsletters for insights that help you meet the challenges of today—and tomorrow.

FDA Updates in Pediatric Care: January 2025

1. PDUFA date set for neffy 1 mg for children who weigh 33 lbs or more

On January 13, 2025, ARS Pharmaceuticals announced the submission of a supplemental New Drug Application to the FDA for a 1 mg dose of neffy (epinephrine nasal spray) for pediatric patients weighing 33 to 66 lbs. Based on pharmacokinetic studies demonstrating comparable epinephrine exposure to intramuscular injections, this expansion follows neffy’s initial FDA approval in August 2024 for patients over 66 lbs. The FDA has set a PDUFA target action date of March 6, 2025, with potential product availability in the second quarter of 2025.

2. FDA bans red no. 3 in food and ingested drugs

On January 15, 2025, the FDA announced the revocation of authorization for FD&C Red No. 3 in food and ingested drugs under the Delaney Clause of the FD&C Act. Based on a 2022 petition citing studies linking the dye to cancer in male rats, manufacturers must reformulate food products by January 15, 2027, and ingested drugs by January 18, 2029. While the FDA maintains that available scientific data do not support human risk, concerns remain regarding potential behavioral effects in children.

3. 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, further broadening access to essential nutritional support.

4. FDA accepts sNDA for higher regimen nusinersen for spinal muscular atrophy

On January 23, 2025, Biogen announced the FDA's acceptance of a supplemental New Drug Application for a higher dose regimen of nusinersen (Spinraza) for spinal muscular atrophy (SMA). Based on data from the phase 2/3 DEVOTE study, the higher dose demonstrated significant improvements in motor function compared to historical controls, with a safety profile consistent with the currently approved 12 mg regimen. If approved, the new regimen could enhance treatment efficacy for SMA patients across all disease types.

5. Apitegromab BLA submitted to FDA for spinal muscular atrophy

On January 29, 2025, Scholar Rock announced the submission of a biologics license application to the FDA for apitegromab, an investigational muscle-targeted therapy for spinal muscular atrophy (SMA). Based on data from the phase 3 SAPPHIRE trial, apitegromab demonstrated a statistically significant 1.8-point improvement in Hammersmith Functional Motor Scale-Expanded scores compared to placebo. Scholar Rock has requested priority review, which, if granted, could accelerate the FDA’s decision timeline to 6 months.

6. FDA approves tocilizumab-anoh as biosimilar for multiple diseases

On January 31, 2025, Celltrion announced the FDA approval of tocilizumab-anoh (Avtozma) as a biosimilar to Actemra, with indications for rheumatoid arthritis, juvenile idiopathic arthritis, giant cell arteritis, and COVID-19. Based on a phase 3 trial demonstrating comparable efficacy, safety, and immunogenicity to reference tocilizumab, the approval includes both intravenous and subcutaneous formulations. This marks Celltrion’s seventh biosimilar approval in the U.S., expanding treatment options for inflammatory diseases.