Denali Therapeutics (Nasdaq: DNLI) announced that the FDA has accepted its Biologics License Application (BLA) for tividenofusp alfa and granted the therapy Priority Review for the treatment of Hunter syndrome, a rare and progressive genetic disorder. The agency set a Prescription Drug User Fee Act (PDUFA) action date of January 5, 2026.
Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is caused by a deficiency of the enzyme IDS, leading to the buildup of toxic substances in the body and brain. Current treatments do not cross the blood-brain barrier and therefore do not address the cognitive and neurological symptoms of the disease.
Tividenofusp alfa is an investigational enzyme replacement therapy designed using Denali’s proprietary TransportVehicle™ platform. It delivers the missing IDS enzyme to both the body and the brain and aims to treat both the physical and cognitive effects of the disease.
“If FDA-approved, tividenofusp alfa would mark the first significant advancement in nearly two decades for enzyme replacement therapy in Hunter syndrome,” said Dr. Carole Ho, Chief Medical Officer at Denali. She called the FDA acceptance “a pivotal milestone” for the company’s platform, which is also being applied to other lysosomal and neurodegenerative diseases.
The BLA is supported by data from Denali’s Phase 1/2 study in 47 patients with Hunter syndrome. Additional evidence will come from the ongoing Phase 2/3 COMPASS trial, designed to support global approvals. Denali is preparing for a potential commercial launch in the U.S. following FDA review.
Street View: BTIG
BTIG analysts reaffirmed their Buy rating and $32 price target following the news. They noted the FDA’s priority designation and acceptance of the application as strong signs of support. “We see this as the FDA being on board,” the note said, adding that Denali’s data showed normalization of molecular markers and clinical improvements such as hearing gains and reduced cognitive decline.
Tividenofusp alfa is also expected to ease patient switching from current therapies, especially in children, due to its improved tolerability and broader tissue delivery. Analysts see the approval as potentially paving the way for other TransportVehicle-based enzyme replacement therapies in diseases like Gaucher and Pompe.
Denali is developing a broad pipeline of blood-brain barrier-penetrant therapies, including additional clinical programs in Sanfilippo Syndrome (DNL126) and Parkinson’s disease (DNL151).
As of the latest update, Denali’s stock (DNLI) was trading modestly lower despite the positive regulatory milestone.
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