Brain HealthFDA Approves First Drug to Treat Neurologic Complications of Hunter Syndrome
The FDA has granted accelerated approval to Avlayah (tividenofusp alfa-eknm), developed by Denali Therapeutics, making it the first treatment approved specifically for the neurologic manifestations of Hunter syndrome (MPS II). Hunter syndrome is a rare X-linked lysosomal storage disorder affecting roughly 500 people in the US, almost exclusively males. Until now, no approved therapy addressed the progressive neurological damage the disease causes. Avlayah is indicated for pediatric patients aged 3 months to 13 years. Approval was based on a surrogate endpoint — significant reduction of cerebrospinal fluid heparan sulfate (CSF HS) — from phase 1/2 trial data. The drug received breakthrough therapy, fast track, priority review, orphan drug, and accelerated approval designations, reflecting the high unmet need in this rare pediatric population.
