Denali Therapeutics Announces FDA Acceptance and Priority Review of Biologics License Application for Tividenofusp Alfa for Hunter Syndrome (MPS II)

Published: 2025-07-07 08:50:29 ET DNLI

Published on 07/07/2025 at 08:50

Denali Therapeutics Inc. 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. If FDA-approved, tividenof Susp 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.

The U.S. Food and drug Administration has granted Fast Track and Breakthrough Therapy designations to tividenofusp alf for development in the treatment of MPS II. In animal models, antibodies and enzymes engineered with the TV platform demonstrate more than 10- to 30-fold greater brain exposure than similar antibodies and enzymes without this technology. Oligonucleotides engineered with the TV platform demonstrate More than a 1,000-fold greater brain exposure in primates than systemically delivered oligonucleotides without this technology.