Table of Contents
The FDA granted, on March 25, 2026, accelerated approval to Denali Therapeutics’ AVLAYAH (tividenofusp alfa-eknm) for the treatment of neurologic manifestations of Hunter syndrome in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment.
AVLAYAH is the first FDA-approved biologic specifically designed to cross the blood-brain barrier and Denali’s first medicine enabled by its TransportVehicle platform.
The approval also carries a Rare Pediatric Disease Priority Review Voucher, which Denali may use to obtain priority review for a future marketing application or transfer to another sponsor.
AVLAYAH will be available in the United States shortly.
In its March 25 announcement, Denali described the approval as the first new FDA-approved treatment option in nearly 20 years for a disease affecting approximately 500 individuals in the United States.
Tividenofusp alfa-eknm is an intravenous enzyme replacement therapy. The approval was based on Phase 1/2 data showing a 91% reduction in cerebrospinal fluid heparan sulfate at Week 24.
The prescribing information warns of hypersensitivity reactions, including anaphylaxis. AVLAYAH is approved as an enzyme replacement therapy for neurologic manifestations of Hunter syndrome in the labeled pediatric population.
Sources: Denali, AVLAYAH Prescribing Information
Comments