FDA news in pediatrics: September 2026

September 2026 brought a dense slate of pediatric-relevant FDA activity, headlined by several first-in-disease approvals for ultra-rare genetic and metabolic conditions: Sanfilippo syndrome type A, Alexander disease, MCT8 deficiency, and ataxia-telangiectasia each gained their first FDA-approved therapy during the month. Treatment options also expanded for spinal muscular atrophy, obstructive hypertrophic cardiomyopathy, and childhood-onset nephrotic syndrome, while several late-stage candidates for MOGAD, hypophosphatasia, and erythropoietic protoporphyria advanced to priority review. Two devices earned breakthrough designations aimed at earlier identification of scoliosis progression and safer insulin dosing in the youngest hospitalized patients.