Inozyme/BioMarin: Enzyme Replacement Therapy Phase 3 Misses Endpoint Inozyme's therapy missed a primary endpoint. BioMarin's $270M investment is at risk. Rare disease treatment faces major setback. EMA has no prior action for this drug. Inozyme's enzyme replacement therapy, partnered with BioMarin, failed to meet one of two dual primary endpoints in a pivotal Phase 3 study. The trial, evaluating the drug in children with a rare genetic disorder, aimed to demonstrate efficacy on two key metrics. While one metric showed improvement, the other, crucial for overall success, did not reach statistical significance. This outcome jeopardizes a potential new treatment for a disease with high unmet medical need. This Phase 3 miss impacts the drug's regulatory pathway. Under FDA and EMA guidelines, dual primary endpoints often require both to be met for approval. Inozyme and BioMarin must now assess if the single met endpoint, alongside secondary data, supports a New Drug Application (NDA) or Marketing Authorization Application (MAA). This situation may necessitate further trials or a re-evaluation of the statistical analysis plan, potentially delaying market entry for years. The setback creates an opening for competitors like Sanofi and Takeda, who are also developing rare disease therapies. BioMarin's substantial $270 million upfront investment is now in jeopardy. This failure could shift focus to other emerging treatments in the rare disease space. A critical board meeting is scheduled for June 15, 2026, to determine the program's future.