Denali Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review its Biologics License Application (BLA) seeking accelerated approval for tividenofusp alfa. This investigational therapy targets Hunter syndrome (MPS II), a rare and progressive genetic disorder.
The FDA granted Priority Review to the BLA, assigning a Prescription Drug User Fee Act (PDUFA) target action date of January 5, 2026. This designation reflects the FDA's recognition of the urgent need for new therapies that could offer significant improvement in the treatment of Hunter syndrome.
The BLA submission is supported by data from an open-label, single-arm Phase 1/2 study of tividenofusp alfa, which involved 47 participants with Hunter syndrome. Tividenofusp alfa is designed to deliver the iduronate 2-sulfatase (IDS) enzyme into both the brain and the body, aiming to address the neurological manifestations of the disease in addition to physical symptoms.
If approved, tividenofusp alfa would represent the first significant advancement in nearly two decades for enzyme replacement therapy for individuals living with Hunter syndrome, due to its potential for delivery to tissues throughout the brain and the body. This acceptance and priority review also mark a pivotal milestone for Denali's proprietary TransportVehicle platform, which continues to advance with the goal of treating a wide range of lysosomal storage diseases and neurodegenerative disorders.
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