BioMarin Presents Positive Data on Voxzogo for Achondroplasia in Children

BioMarin Pharmaceutical reported data showing the benefits of Voxzogo, a once-daily injection for treating achondroplasia, in children. The company's commercial products include Vimizim, Naglazyme, Palynziq, Brineura, Aldurazyme, Roctavian, Kuvan, and Voxzogo, which address various genetic conditions.

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has announced new data from 14 studies presented at the American Society for Bone and Mineral Research 2025 (ASBMR) Annual Meeting in Seattle. The studies include results demonstrating the treatment with VOXZOGO® (vosoritide) led to anatomical improvements in spinal morphology in young children and continued efficacy in children who received the medicine after the onset of puberty.

The data, presented in a randomized, double-blind, placebo-controlled Phase 2 CANOPY clinical study, showed that children (n=40) who received VOXZOGO for 52 weeks experienced measurable improvement in interpedicular distance (IPD) and greater spinal canal width in all five vertebrae in the lower back (L1 through L5) compared to those who received placebo (n=27). Additionally, 57% of children who received VOXZOGO demonstrated a reduction in thoracolumbar kyphosis (TLK) compared to 33% of those who received placebo.

In an open-label, Phase 3 CANOPY long-term extension study, teenagers with achondroplasia continued to benefit from VOXZOGO after puberty onset. Young men who received VOXZOGO experienced 24.62 cm of growth from the mean age of puberty onset until age 18, compared to 17.07 cm in untreated individuals, a difference of 7.55 cm. For young women, growth from the mean age of puberty until age 16 was 21.20 cm compared to 13.13 cm in untreated individuals, a difference of 8.07 cm.

BioMarin also presented research from four studies reflecting its leadership in deepening the scientific understanding of and highlighting the unmet medical needs for people with ENPP1 deficiency, a rare, serious, and progressive genetic condition that affects blood vessels, soft tissues, and bones. The company is advancing BMN 401, a potential first-in-disease enzyme therapy for the condition, with initial pivotal data anticipated in the first half of 2026.

These findings reinforce BioMarin's position as a leader in the development of therapies for rare genetic conditions, with a focus on improving the quality of life for patients and their families.

References:
[1] https://finance.yahoo.com/news/biomarin-shares-data-reinforcing-leadership-130000693.html