BioMarin’s Phase 3 program for its ENPP1 deficiency therapy landed below regulatory expectations, missing a key goal in a rare inherited enzyme deficiency study. The results add uncertainty around the company’s approval prospects for this treatment. BioMarin previously acquired the asset via its 2025 purchase of Inozyme Pharma, and the latest readout reinforces how fragile rare-disease pipelines can be when endpoints are narrowly missed. While the company may continue to evaluate next steps, investors typically reassess development timelines and future capital allocation immediately after pivotal failures.