FDA approved levacetylleucine (Aqneursa) for ataxia in ataxia-telangiectasia, the first therapy for the disease, in patients 15 kg or heavier.
News|Articles|June 5, 2026
Pediatric clinical trial update: May 2026
Author(s)Morgan Ebert, Executive Editor
Fact checked by: Benjamin P. Saylor
A look back at the latest clinical trial updates for the pediatric population during May 2026.
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May brought a steady stream of clinical trial developments with potential implications for pediatric care, spanning infectious diseases, neurology, endocrinology, gastroenterology, and rare diseases. From new data on antiviral prevention strategies and emerging therapies for Tourette syndrome to advances in diabetes management and treatments for pediatric cardiomyopathy, investigators reported findings that may help shape future clinical practice and regulatory decisions.
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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.
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.
XOCOVA gives pediatricians a new oral option to help prevent COVID-19 after exposure in adolescents aged 12 years and older.
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