Denali Therapeutics Inc. announced U.S. FDA accelerated approval of AVLAYAH™ (tividenofusp alfa-eknm) for neurologic manifestations in presymptomatic or symptomatic pediatric patients weighing at least 5 kg with Hunter syndrome (MPS II) prior to advanced neurologic impairment. This marks the first FDA-approved therapy designed to cross the blood-brain barrier using the company's TransportVehicle™ platform.
Key takeaways
U.S. FDA granted accelerated approval for AVLAYAH on March 25, 2026.
AVLAYAH indicated for neurologic manifestations of Hunter syndrome (MPS II) in pediatric patients ≥5 kg prior to advanced impairment.
Phase 1/2 trial showed 91% reduction (95% CI: 89%, 92%) in CSF HS levels by week 24; 93% of treated patients had normal levels.
First new FDA-approved treatment for Hunter syndrome in nearly 20 years.
FDA awarded Rare Pediatric Disease Priority Review Voucher (PRV) in connection with approval.
Ongoing Phase 2/3 COMPASS study to confirm clinical benefit.