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Denali Therapeutics Announces U.S. FDA Approval of AVLAYAH™ (tividenofusp alfa-eknm) for Treatment of Hunter Syndrome (MPS II)

DNLI
  • FDA granted accelerated approval for AVLAYAH (tividenofusp alfa-eknm), the first FDA-approved treatment for Hunter syndrome (MPS II) in nearly 20 years.
  • AVLAYAH is the first FDA-approved medicine in a new class of biotherapeutics that use the transferrin receptor to cross the blood-brain barrier.
  • The drug is Denali's first therapy enabled by its TransportVehicle™ platform, designed to deliver biotherapeutics to the whole body, including the brain.
  • Approval is indicated for pediatric patients weighing at least 5 kg with Hunter syndrome prior to advanced neurologic impairment.
  • Continued approval is contingent upon verification of clinical benefit in the ongoing Phase 2/3 COMPASS confirmatory trial.
  • FDA approval was based on a 91% reduction in cerebrospinal fluid heparan sulfate (CSF HS) levels, a biomarker surrogate endpoint.
  • A Rare Pediatric Disease Priority Review Voucher (PRV) was awarded to Denali Therapeutics in connection with this approval.
  • AVLAYAH is an enzyme replacement therapy composed of the IDS enzyme fused to Denali's TransportVehicle™ platform.
  • The medication is administered via intravenous infusion once weekly and will be available in the U.S. shortly after approval.
  • The Phase 1/2 trial showed 93% of treated patients had CSF HS levels within the range of individuals without Hunter syndrome at week 24.
  • Most common adverse reactions include infusion-related reactions, upper respiratory infections, fever, anemia, cough, vomiting, diarrhea, rash, and headache.
  • Serious side effects may include hypersensitivity reactions (anaphylaxis), infusion-associated reactions, anemia, and membranous nephropathy.
  • Denali Therapeutics will host a conference call and webcast at 12:30 p.m. Eastern time on March 25, 2026, to discuss the approval.