What happened?
The FDA approved tividenofusp alfa-eknm (Avlayah) for neurological manifestations of Hunter syndrome when started before advanced neurological impairment in eligible children. The accelerated approval was based on reduction of cerebrospinal-fluid heparan sulfate, a surrogate biomarker considered reasonably likely to predict benefit.
Why does it matter?
Hunter syndrome can damage the brain as well as multiple organs. A therapy engineered to address central nervous system disease represents a major conceptual advance for this inherited lysosomal disorder.
How to interpret the evidence
Because the approval is accelerated and biomarker-based, a randomized confirmatory trial is required to establish the magnitude of clinical neurological benefit.
What remains uncertain?
It does not reverse advanced neurological damage and it is not a cure for all manifestations of Hunter syndrome.
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