Denali announces FDA acceptance, priority review for tividenofusp alfa BLA

Denali Therapeutics (DNLI) announced that the U.S. Food and Drug Administration has accepted for review the Biologics License Application seeking accelerated approval for tividenofusp alfa for the treatment of Hunter syndrome, a rare and progressive genetic disorder. The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act target action date of January 5, 2026. Hunter syndrome is caused by a deficiency in the iduronate 2-sulfatase 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. The BLA submission is supported by data from the open-label, single-arm Phase 1/2 study of tividenofusp alfa in 47 participants with Hunter syndrome. Denali continues to prepare for a potential commercial launch in the U.S. and is conducting the ongoing Phase 2/3 COMPASS study to support global regulatory approvals.