BioMarin’s ENDO 2026 data show VOXZOGO sustained 3-year growth in hypochondroplasia; BMN 333 supports weekly dosing in achondroplasia
Written By: Meghana Jinka, PharmD
Reviewed By: Pharmacally Editorial Team
BioMarin Pharmaceutical reported new clinical data at ENDO 2026 highlighting continued progress for its skeletal growth disorder portfolio. Long-term results from an investigator-sponsored Phase 2 extension study showed that VOXZOGO (vosoritide) maintained growth benefits over three years in children with hypochondroplasia, while Phase 1 findings for investigational BMN 333 supported advancement into late-stage development for achondroplasia.
The VOXZOGO data add to growing evidence supporting expansion of the therapy beyond its current use in achondroplasia.
BioMarin recently reported positive topline results from the pivotal Phase 3 CANOPY-HCH-3 study (NCT06455059) and plans to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration in the third quarter of 2026.
Long-Term VOXZOGO Data Show Sustained Growth Gains
Hypochondroplasia is a rare inherited skeletal dysplasia caused primarily by mutations in the FGFR3 gene, leading to impaired bone growth, disproportionate short stature, and potential neurological and orthopedic complications. No therapies are currently approved by the FDA or European Medicines Agency for the condition.
The three-year extension study (NCT04219007) evaluated 13 children with hypochondroplasia treated with VOXZOGO, a C-type natriuretic peptide (CNP) analog that counteracts excessive FGFR3 signaling and promotes bone growth.
Investigators observed sustained improvements in both annualized growth velocity (AGV) and height standard deviation score (SDS) throughout treatment. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year after one year (p<0.001) and remained above baseline levels through years two and three. Mean height SDS improved by 0.72 standard deviations over the three-year treatment period.
Researchers also reported a favorable safety profile, with no new safety concerns emerging during long-term follow-up.
According to BioMarin Chief Research and Development Officer Greg Friberg, the findings reinforce the positive Phase 3 results recently reported in hypochondroplasia and provide additional evidence that VOXZOGO may deliver durable growth benefits in this underserved patient population.
BMN 333 Supports Potential Weekly Dosing Strategy
BioMarin also presented new Phase 1 data for BMN 333, a long-acting CNP therapy under development for achondroplasia, the most common form of skeletal dysplasia associated with disproportionate short stature.
Unlike daily VOXZOGO injections, BMN 333 is being developed as a potential once-weekly treatment. In a single-ascending-dose study involving healthy adult volunteers, BMN 333 achieved sustained systemic exposure and prolonged pharmacodynamic target engagement consistent with weekly administration.
At the highest evaluated dose of 500 μg/kg, BMN 333 increased exposure to free CNP more than 13-fold compared with another long-acting CNP agent. The therapy was well tolerated across all tested dose levels, with no dose-limiting toxicities or treatment-related serious adverse events reported.
Path Toward Approval and Expansion
BioMarin began enrolling patients in a registration-enabling Phase 2/3 study of BMN 333 in April 2026. The company expects to provide an update from the dose-finding portion of the trial in 2027.
For VOXZOGO, the upcoming FDA submission represents the next major milestone in potentially becoming the first approved treatment for hypochondroplasia. If approved, the therapy could address a significant unmet need for an estimated 14,000 eligible children across BioMarin’s global footprint.
What This Means for Patients
The new findings offer encouraging news for families affected by hypochondroplasia and achondroplasia, two rare genetic conditions that impair normal bone growth. For children with hypochondroplasia, long-term VOXZOGO treatment continued to improve growth over three years while maintaining a favorable safety profile. There are currently no approved medicines for hypochondroplasia in the United States or Europe, making BioMarin’s planned FDA submission a potentially significant step toward the first approved treatment for the condition.
For children with achondroplasia, the early BMN 333 results suggest that effective growth-promoting therapy may one day be delivered through once-weekly injections rather than daily treatment. If future studies confirm its benefits, BMN 333 could offer a more convenient treatment option while maintaining strong biological activity and safety.
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About the Writer
Meghana Jinka (LinkedIn) is a Pharm.D graduate with a strong interest in clinical pharmacy, clinical research, pharmacovigilance, and medical writing. She has developed expertise in evaluating scientific literature, interpreting clinical data, and communicating complex medical information in a clear and accessible manner. Through clinical training, patient counseling, and healthcare awareness activities, she has gained practical experience in evidence-based medicine and patient-centered care. Passionate about healthcare communication, Meghana is committed to developing accurate, engaging, and evidence-based healthcare documents that support healthcare professionals and the wider community.