Core Points - Denali Therapeutics Inc. announced an extension of the FDA review timeline for the Biologics License Application (BLA) for tividenofusp alfa from January 5, 2026, to April 5, 2026 [1][2] - The extension is due to Denali's submission of updated clinical pharmacology information, classified as a Major Amendment, and does not relate to efficacy, safety, or biomarkers [2] - Denali is preparing for the potential approval and commercial launch of tividenofusp alfa, emphasizing the urgency to deliver this therapy to the MPS community [3] Product Information - Tividenofusp alfa (DNL310) is designed to deliver the iduronate 2-sulfatase (IDS) enzyme to address symptoms of Hunter syndrome, including behavioral, cognitive, and physical symptoms [4] - The FDA has granted Fast Track and Breakthrough Therapy designations to tividenofusp alfa, and the European Medicines Agency has granted Priority Medicines designation [4] - The Phase 2/3 COMPASS study is currently enrolling participants in North America, South America, and Europe to support global approval [5] Disease Background - Hunter syndrome (MPS II) is a rare genetic lysosomal storage disease caused by mutations in the IDS gene, leading to a deficiency of the IDS enzyme [6] - Symptoms include developmental delays, cognitive decline, and physical complications, with current therapies not addressing neurological symptoms due to their inability to cross the blood-brain barrier [6] - There is a significant unmet need for therapies that can address both central nervous system and peripheral manifestations of Hunter syndrome [6] Company Overview - Denali Therapeutics is focused on developing therapies that can cross the blood-brain barrier for neurodegenerative and lysosomal storage diseases [7] - The company employs a rigorous approach to assess genetically validated targets and develop treatments guided by biomarkers [7]