Summary
AVLAYAH (tividenofusp alfa‑eknm) has received FDA accelerated approval as the first Hunter syndrome therapy specifically designed to treat neurologic manifestations by crossing the blood–brain barrier.
The drug uses Denali’s TransportVehicle platform to fuse the IDS enzyme with a transferrin‑receptor–targeting component, enabling enzyme delivery to both brain and peripheral tissues with a single weekly IV infusion.
Approval is based on Phase 1/2 data showing deep reductions (around 90%) in cerebrospinal fluid heparan sulfate and normalization of this key biomarker in most treated children, a change judged reasonably likely to predict neurologic benef...
