BioMarin’s Phase 3 program for a rare inherited enzyme deficiency drug (from its Inozyme Pharma acquisition) missed the mark, meeting only one of two primary goals. The company’s results were characterized as a mixed outcome in coverage of the late-stage dataset. The Phase 3 performance matters because the indication has no FDA-approved therapies, which raises the bar for statistical and clinical impact in order to support regulatory review. The development therefore introduces additional uncertainty around potential approval prospects for this specific biologics strategy. The readout also reinforces how rare-disease investors weigh endpoint selection and robustness across primary goals when single-readout success is required.