The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, as the first treatment for pediatric MPS IIIA (Sanfilippo syndrome type A).

FDA approved apitegromab-mstn (Isembyld) for SMA in patients 2 years and up, based on phase 3 data showing added motor gains with SMN2 therapy.

Week 52 data from the reACHin trial show navepegritide stabilized foramen magnum stenosis and boosted growth in infants with achondroplasia.

Phase 3 CANOPY-HCH-3 data show vosoritide significantly improved growth velocity, height, and arm span in children with hypochondroplasia.

A recap of the top FDA approvals, authorizations, and regulatory decisions affecting pediatric care during August 2026.

The FDA approved zilganersen (Zanvastro) for Alexander disease in patients from infancy through adulthood, based on Phase 1-3 gait speed data.

Infliximab or steroids may be more effective than anakinra as adjunctive therapy for children with MIS-C who do not respond to IVIG.

FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.