Top pediatric FDA approvals of 2025

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

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.

The FDA granted priority review to efzimfotase alfa for HPP in patients aged 2 years and older; a decision is expected in the first half of 2027.

Adolescent VALIANT subgroup data show pegcetacoplan cut proteinuria 75% vs placebo, supporting its expanded pediatric kidney indication.

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).

Aquestive resubmitted its NDA for dibutepinephrine sublingual film Anaphylm, citing new human factors and PK data addressing the FDA's CRL.

Phase 3 CROSSING trial data show tezepelumab improved histologic remission and dysphagia versus placebo through week 52 in EoE.