Overview
- BioMarin reported Wednesday that its 80‑patient late‑stage trial showed children treated with Voxzogo grew 2.33 cm more than placebo in annualized growth rate after 52 weeks.
- The study also delivered statistically significant gains in standing height and arm span, key secondary measures that reflect functional growth beyond speed of growth.
- BioMarin said it will submit a supplemental application to U.S. regulators in the third quarter of 2026 and then pursue filings in Europe and other regions to expand Voxzogo’s use.
- The results prompted a positive investor response, lifting BioMarin shares in extended trading as investors weighed the drug’s commercial potential for a currently untreated rare disease.
- Voxzogo is already approved in the U.S. for achondroplasia, and if regulators accept the new data it could become the first approved therapy for hypochondroplasia, though agencies will review the size of the benefit and longer‑term safety and growth follow‑up.