Top pediatric FDA approvals of 2025

FDA approved Lupin's generic of Firdapse for LEMS in patients 6 years and older, but a patent settlement delays US marketing until at least 2035.

A recap of the top pediatric clinical trial results and pipeline data readouts during October 2026.

A recap of the top FDA approvals, regulatory decisions, and pipeline updates affecting pediatric care during October 2026.

In PROGRESS Cohort 2, zilurgisertib reduced total HO volume vs placebo at 24 weeks in children aged 6 to 11 with FOP, with no new HO lesions.

Registry data presented at ASN Kidney Week 2026 linked SCD-PED to higher 90-day survival vs historical controls in pediatric sepsis-associated AKI.

The FDA approved insulin icodec-abae for children 10+ with T2D, based on adult ONWARDS data and a single-dose pediatric pharmacokinetic study.

A Contemporary Pediatrics Peer Exchange panel reviews RSV risk, testing, maternal vaccination, and long-acting monoclonal antibodies, and where infants still slip through the cracks.

FDA approves berotralstat 150-mg oral pellets for HAE prophylaxis in patients 12+ who cannot or prefer not to swallow capsules.