Denali Therapeutics announced that the US Food and Drug Administration (FDA) has accepted and granted priority review to its Biologics License Application (BLA) for tividenofusp alfa, a potential enzyme replacement therapy for Hunter syndrome (MPS II). According to the company, the target action date under the Prescription Drug User Fee Act (PDUFA) is January 5, 2026.
The company said that tividenofusp alfa is designed to cross the blood-brain barrier and address both central nervous system and peripheral manifestations of Hunter syndrome, a rare and progressive genetic disorder. The BLA submission is based on results from the Phase 1/2 and ongoing Phase 2/3 clinical studies, which the company claims have shown reductions in cerebrospinal fluid glycosaminoglycans and positive trends in neurocognitive function.
The FDA previously granted tividenofusp alfa both Breakthrough Therapy and Rare Pediatric Disease designations, which, according to the company, reflect the significant unmet need in Hunter syndrome treatment.
Denali Therapeutics said that if approved, tividenofusp alfa would be the first therapy targeting both brain and body symptoms of Hunter syndrome. The company plans to collaborate closely with the FDA during the review process to bring the treatment to patients as soon as possible.
