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Denali Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application (BLA) for Tividenofusp Alfa for Hunter Syndrome (MPS II)
FDA assigns PDUFA target action date of January 5, 2026, for decision on accelerated approvalTividenofusp alfa is designed to deliver missing enzyme to entire
About this update from Denali Therapeutics Inc.
[{"type":"text","content":"FDA assigns PDUFA target action date of January 5, 2026, for decision on accelerated approvalTividenofusp alfa is designed to deliver missing enzyme to entire body and cross blood-brain barrier into the brainTividenofusp alfa leads company’s broader TransportVehicle™-enabled pipeline SOUTH SAN FRANCISCO, Calif., July 07, 2025 (GLOBE NEWSWIRE) -- Denali Therapeutics Inc. (Nasdaq: DNLI) today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the Biologics License Application (BLA) seeking accelerated approval for tividenofusp alfa for the treatment of Hunter syndrome (mucopolysaccharidoses type II, or MPS II), a rare and progressive genetic disorder. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026. Hunter syndrome is caused by a deficiency in the iduronate 2-sulfatase (IDS) enzyme, which is needed to break down complex sugars called glycosaminoglycans that build up in the brain and body, starting at a young age. Current therapies do not cross the blood-brain barrier and lack the potential to address the impact of the disease on cognitive abilities and behavior. Tividenofusp alfa is an investigational, next-generation enzyme replacement therapy composed of IDS fused to Denali’s TransportVehicle™ platform and is designed to deliver IDS into the brain and the body, aiming to treat neurological manifestations of the disease in addition to physical symptoms. “We are grateful to the FDA for their recognition of the urgent need for new therapies that could offer a significant improvement in the treatment of Hunter syndrome, as reflected by their priority review designation for our Biologics License Application for tividenofusp alfa,” said Carole Ho, M.D., Chief Medical Officer and Head of Development of Denali Therapeutics. “If FDA-approved, tividenofusp alfa would mark the first significant advancement in nearly two decades for enzyme replacement therapy for individuals living with Hunter syndrome because of its potential for delivery to tissues throughout the brain and the body. This is also a pivotal milestone for our TransportVehicle platform, which continues to progress with the aim of treating a wide range of lysosomal storage diseases and neurodegenerative disorders.” The BLA submission is supported by data from the open-la...