BioMarin Pharmaceutical (BMRN) said Tuesday it presented new clinical data for its Voxzogo and BMN 333 treatments targeting hypochondroplasia and achondroplasia, both of which are growth-related bone conditions.
An investigator-led Phase 2 extension study showed that Voxzogo led to sustained growth improvements in children with hypochondroplasia, the company said.
The company intends to submit a supplemental New Drug Application to the US Food and Drug Administration during Q3 based on a separate Phase 3 study, it said.
Initial Phase 1 testing of BMN 333 in healthy adults demonstrated prolonged drug exposure supporting a weekly dosing schedule for achondroplasia, BioMarin Pharmaceutical said.
Patient enrollment for a Phase 2/3 study of the weekly drug began in April, with a data update from the dose-finding segment expected in 2027, the company added.
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