BioMarin shares VOXZOGO three-year data and BMN 333 Phase 1 results

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented new clinical data on VOXZOGO (vosoritide) in hypochondroplasia and its investigational drug BMN 333 in achondroplasia at ENDO 2026, the Endocrine Society Annual Meeting in Chicago, according to a company press release.

A Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. at Children's National Hospital, showed mean height standard deviation score improved by 0.72 SD over three years. Mean annualized growth velocity increased from 4.27 cm/year at baseline to 7.24 cm/year at year one and remained above baseline through years two and three. No significant safety concerns were reported.

BioMarin said it plans to submit a supplemental New Drug Application to the U.S. Food and Drug Administration for VOXZOGO in hypochondroplasia in the third quarter of 2026, based on results from its CANOPY-HCH-3 Phase 3 pivotal study. No medicines are currently approved by the FDA or European Medicines Agency for hypochondroplasia.

Separately, Phase 1 data for BMN 333, a long-acting C-type natriuretic peptide candidate for achondroplasia, showed sustained systemic exposure in healthy adults following a single ascending dose, with results supporting a weekly dosing schedule. The maximum examined dose of 500 μg/kg increased exposure to free CNP by more than 13 times compared to another long-acting CNP agent. No dose-limiting toxicities or treatment-related serious adverse events were reported.

BioMarin began enrolling patients in a registration-enabling Phase 2/3 study of BMN 333 in April. A data update from the dose-finding segment is expected in 2027.

You May Also Be Interested In

• Alphabet Sees FY26 Capex $195B To $205B, Saw $180B To $190B
• Dexcom selected as first participant in FDA's TEMPO pilot
• Penumbra study shows THUNDERBOLT met stroke treatment goals