BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced new data from studies of VOXZOGO® (vosoritide) in children with hypochondroplasia and the investigational medicine BMN 333 in achondroplasia were presented at ENDO 2026, the Endocrine Society Annual Meeting, in Chicago.

New data from a Phase 2, investigator-sponsored three-year extension study of VOXZOGO in 13 children with hypochondroplasia, led by Andrew Dauber, M.D. and investigators at Children's National Hospital, demonstrated sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS), with a favorable safety profile. Mean height SDS improved by 0.72 SD over three years of treatment, while mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year at year one (p<0.001) and remained above baseline through years two and three.

BioMarin recently announced positive topline results from CANOPY-HCH-3, its registration-enabling Phase 3 pivotal study evaluating VOXZOGO in children with hypochondroplasia. These results will be included in the supplemental New Drug Application submission to the U.S. Food and Drug Administration planned for the third quarter of 2026.

"Building on the excellent Phase 3 results we recently announced, these new longer-term data further reinforce the potential of VOXZOGO to meaningfully improve growth outcomes for children with hypochondroplasia," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "Importantly, we continue to observe sustained growth improvements over time and a favorable safety profile, adding to the growing body of evidence supporting VOXZOGO for this potential new indication."

New BMN 333 Phase 1 Data Support Advancement Into Late-Stage Development

Additional data being presented at ENDO 2026 highlight the potential of investigational BMN 333, BioMarin's long-acting C-type natriuretic peptide (CNP) for achondroplasia. In a Phase 1 single-ascending dose study in healthy adults, BMN 333 demonstrated sustained systemic exposure associated with prolonged pharmacodynamic target engagement, supporting a weekly dosing schedule. The maximum examined dose of BMN 333 (500 μg/kg) increased exposure to free CNP by more than 13 times compared to another long-acting CNP agent, reflecting the potential of BMN 333 to become the new standard of care in achondroplasia. BMN 333 was well tolerated across all dose levels evaluated, with no dose-limiting toxicities or treatment-related serious adverse events.

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

Below are key presentations for achondroplasia and hypochondroplasia at ENDO, with all times listed in Central Daylight Time:

Vosoritide Treatment in Children With Hypochondroplasia: Three-Year Results From a Phase 2 Extension Trial

Oral Presentation #ORF47-08

Monday, June 15, 3 – 3:15 p.m.

BMN 333 Achieves High Sustained Released Vosoritide Exposure With Favorable Safety: Phase 1 Results That Support Phase 2/3 Trials in Achondroplasia

Poster Presentation #SUN-212

Sunday, June 14, 9 a.m. – 4 p.m.

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