BioMarin Pharmaceutical Inc. presented data at The Endocrine Society Annual Meeting, ENDO 2022, demonstrating the Company’s ongoing commitment to understanding the lifetime impact of achondroplasia, the most common form of disproportionate short stature. The Company provided data from a Phase 2 randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of VOXZOGO™ (vosoritide) for Injection in infants and children aged 0 to less than five years old.
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In the overall population of all randomized and sentinel subjects, the study showed an improvement in height Z-score (a measure of height adjusted for age and sex in reference to the average stature population and reported as a Standard Deviation, SD) as compared to placebo at 52 weeks. Voxzogo (n=43), compared to placebo (n=32), increased height Z-score by 0.30 SD (95% CI 0.07, 0.54) and annualized growth velocity (AGV) by 0.92cm/year (95% CI 0.24, 1.59). This improvement in height Z-score was consistent with improvements previously observed after one year of treatment in children over five years of age. In the randomized population (excluding sentinel subjects) and in the individual age subgroups, a trend toward increased height Z-score was observed. Voxzogo did not significantly impact upper-to-lower body segment ratio, which changed by -0.06 (95% CI -0.15, 0.03) over this 52-week period.
“We are pleased by these 52-week analysis results showing positive changes with Voxzogo compared to placebo in height Z-score and AGV,” said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. “We look forward to discussing next steps regarding our efforts to expand access to Voxzogo treatment for this younger age group. We remain grateful to the children and families participating in the clinical study program.”
“These data are very encouraging and supportive of early treatment initiation – there is clearly the potential these younger children could see meaningful benefit from treatment with Voxzogo. Importantly this is just the beginning: we look forward to the results of future analyses addressing additional endpoints and await further data on longer term follow up in this population with complex needs,” said Melita Irving, Clinical Geneticist at Guy’s and St Thomas’ NHS Foundation Trust, London, UK and investigator for the Voxzogo clinical program at the Evelina London Children’s Hospital.