Denali Therapeutics (NASDAQ:DNLI), a biopharmaceutical company dedicated to developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB), announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for AVLAYAH™ (tividenofusp alfa-eknm).
AVLAYAH is indicated for the treatment of neurologic manifestations of Hunter syndrome (mucopolysaccharidosis type II, or MPS II) in pediatric patients weighing at least 5 kg who have not yet reached advanced neurologic impairment.
The approval represents a historic milestone as the first FDA-approved medicine to utilize a transferrin-receptor (TfR) mediated delivery mechanism to cross the blood-brain barrier.
While existing enzyme replacement therapies (ERTs) effectively manage the physical symptoms of Hunter syndrome, they typically cannot reach the central nervous system.
AVLAYAH, administered once weekly via intravenous infusion, is specifically designed to address both the systemic and devastating neurologic aspects of the disease.
The FDA’s decision was based on a surrogate biomarker endpoint: a 91% mean reduction in cerebrospinal fluid heparan sulfate (CSF HS) by week 24 of treatment.
This reduction in the buildup of toxic sugar molecules is considered reasonably likely to predict clinical benefit in preventing or slowing neurocognitive decline.
As part of the accelerated approval pathway, continued approval may be contingent upon the verification of clinical benefit in the ongoing Phase 2/3 COMPASS trial.
In recognition of the drug's potential for a rare pediatric disease, the FDA awarded Denali a Rare Pediatric Disease Priority Review Voucher (PRV) upon approval.
These vouchers are highly valuable assets in the biotechnology sector, as they can be used to accelerate the review of a future drug application or sold to another manufacturer.
Denali is preparing for an immediate commercial launch in the United States, supported by a robust supply chain and patient advocacy network.