Aquestive resubmitted its NDA for dibutepinephrine sublingual film Anaphylm, citing new human factors and PK data addressing the FDA's CRL.
News|Articles|April 1, 2025
FDA news in pedatrics: March 2025
Author(s)Joshua Fitch, Senior Editor
A look back at the FDA submissions and regulatory decisions in the pediatric health care space from March 2025.
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Another month has come and gone, and with it, several FDA approvals and regulatory updates related to the ever-evolving landscape of pediatric health care.
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Lavengratinib increased annualized height velocity in 7 children with achondroplasia, but open-label phase 2 findings remain preliminary.
The FDA granted satralizumab priority review for MOGAD after phase 3 findings showed a 68% reduction in relapse risk versus placebo.
Amélie Lothe, PhD, discusses findings showing early, sustained benefits of fenfluramine treatment for patients with Lennox-Gastaut syndrome.
The FDA accepted the dersimelagon NDA with priority review for EPP and XLP; a decision on the oral MC1R agonist is expected by February 2027.
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.
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