BioMarin Unveils Promising New Data on VOXZOGO and BMN 333 at ENDO 2026

BioMarin Unveils New Data on VOXZOGO and BMN 333 at ENDO 2026

BioMarin Pharmaceutical Inc. has recently disclosed insightful findings regarding their treatments VOXZOGO® (vosoritide) and investigational BMN 333 during the ENDO 2026 Annual Meeting in Chicago. These studies focused on conditions affecting children's growth, specifically hypochondroplasia and achondroplasia, two forms of skeletal dysplasia associated with short stature.

Sustained Growth Improvements from VOXZOGO

The spotlight on VOXZOGO revealed compelling results from a three-year Phase 2 study that examined its effectiveness in children diagnosed with hypochondroplasia. Conducted by Dr. Andrew Dauber and his team at Children’s National Hospital, the study highlighted sustained improvements in annualized growth velocity (AGV) and height standard deviation score (SDS). The average height SDS increased by 0.72 SD, reflecting significant advancements in growth over the treatment period.

Notably, the mean AGV surged dramatically—from 4.27 cm/year at baseline to an impressive 7.24 cm/year in the first year, maintaining levels above the baseline through the subsequent two years. This long-term follow-up presents an optimistic outlook for VOXZOGO as a potential breakthrough in treating hypochondroplasia, a rare condition that lacks FDA-approved therapeutic options.

In light of these results, BioMarin is gearing up to submit a supplemental New Drug Application to the U.S. Food and Drug Administration (FDA) later this year, building on the success of their recent Phase 3 pivotal study CANOPY-HCH-3.

BMN 333: A Potential New Standard in Treating Achondroplasia

The presentation at ENDO 2026 also included early findings for BMN 333, a new treatment being assessed for children with achondroplasia. This investigational medicine, which leverages the C-type natriuretic peptide (CNP) pathway, showed promise in a Phase 1 trial conducted with healthy adults. Results indicated that BMN 333 supports a weekly dosing schedule due to its prolonged pharmacodynamic target engagement. The study's findings suggest that it could significantly elevate the standard of care for achondroplasia, a condition affecting millions globally.

The Phase 1 trial revealed that the highest tested dose of BMN 333 dramatically increased systemic CNP levels, thereby supporting its potential efficacy. The treatment was well tolerated, with no significant side effects reported. BioMarin has now initiated patient enrollment for a Phase 2/3 registration-enabling study, indicating strong forward momentum for this investigational treatment.

Understanding Skeletal Dysplasia

Skeletal dysplasias, such as achondroplasia and hypochondroplasia, are genetic conditions that lead to abnormalities in bone growth and structure, resulting in disproportionate short stature and various physical complications. Achondroplasia, the more common of the two, affects approximately one in 25,000 live births, typically emerging due to a spontaneous mutation in the FGFR3 gene. On the other hand, hypochondroplasia is characterized by similar growth impairment, but with a broader clinical spectrum impacting physical functionalities.

BioMarin estimates that around 14,000 children with hypochondroplasia may be suitable candidates for VOXZOGO treatment, thus highlighting the urgent need for effective therapies in this field.

Conclusion

The data presented by BioMarin at ENDO 2026 marks a significant advancement in the treatment landscape for conditions associated with short stature. The sustained growth improvements seen in VOXZOGO and promising Phase 1 results for BMN 333 illuminate a hopeful path forward for families affected by these rare disorders. As BioMarin continues its clinical trials, the biomedical community eagerly anticipates further developments that could redefine standards of care in skeletal dysplasia.