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Denali Therapeutics Announces U.S. FDA Approval of AVLAYAH™ (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II)
First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved medicine in emerging
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"First new FDA-approved treatment option in nearly 20 years for families living with this rare lysosomal storage diseaseFirst FDA-approved medicine in emerging new class of biotherapeutics that leverage transferrin receptor to cross blood-brain barrierDenali’s first medicine enabled by its TransportVehicle™ platform designed to deliver biotherapeutics to whole body, including brainRare Pediatric Disease Priority Review Voucher (PRV) awarded in connection with FDA approvalDenali to host conference call and webcast today at 12:30 p.m. Eastern time SOUTH SAN FRANCISCO, Calif., March 25, 2026 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced the U.S. Food and Drug Administration (FDA) has granted accelerated approval for AVLAYAH™ (tividenofusp alfa-eknm), the first FDA-approved biologic specifically designed to cross the blood-brain barrier and reach the whole body, including the brain. AVLAYAH is an enzyme replacement therapy indicated for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurologic impairment. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial. “The approval of AVLAYAH is a new era for the Hunter syndrome community as we deliver the first FDA-approved therapy designed to cross the brain’s protective barrier for individuals and families living with this debilitating disease. This approval reflects the determination and partnership of the MPS community, as well as the FDA’s collaborative engagement to incorporate biomarker evidence to help accelerate the development of urgently needed treatments,” said Ryan Watts, Ph.D., co-founder and Chief Executive Officer of Denali Therapeutics. “This milestone validates our TransportVehicle platform and its potential to overcome the long-standing challenge of delivering biologic medicines across the blood-brain barrier, with the aim to transform the treatment of a wide range of neurodegenerative diseases, lysosomal storage disorders and other serious diseases that impact millions worldwide.\" Hunter syndrome is a rare genetic disease caused by a deficiency in the iduronate 2-sulfatase (IDS) enzyme, which is needed to break dow...