BioMarin moved to discontinue BMN401, an enzyme replacement therapy for ENPP1 deficiency, after previously reporting mixed Phase 3 results and choosing not to make an immediate snap decision. The company ultimately ended development across all indications, completing a short-lived expansion attempt that followed its July 2025 acquisition of Inozyme Pharma for about $270 million. The decision effectively marks the end of BMN401 as the acquired assets’ core contributor, with investors having expected the discontinuation after the mixed late-stage readouts. BioMarin’s commercial portfolio is now expected to carry the business while management redeploys growth capital toward other pipeline augmentation. BioMarin’s action also highlights the heightened post-acquisition execution risk for rare-disease assets once efficacy signals fail to deliver a clean confirmatory profile.