The FDA approved Avlayah (tividenofusp alfa-eknm), the first drug specifically targeting neurologic complications of Hunter syndrome in pediatric patients aged 3 months to 13 years. Hunter syndrome is a rare X-linked genetic disorder affecting approximately 500 people in the US, almost exclusively males. The approval was based on accelerated approval pathway using reduced cerebrospinal fluid heparan sulfate as a surrogate endpoint. This represents a significant breakthrough for families dealing with this devastating rare disease that causes progressive neurological decline.
