U.S. Food and Drug Administration Approves BioMarin’s VOXZOGO® (vosoritide) for Children Under 5 Years with Achondroplasia

Expanded Indication in the U.S. Now Includes Children of all ages with Achondroplasia

BioMarin Pharmaceutical, a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the U.S. Food and Drug Administration (FDA) has approved the supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) to increase linear growth in pediatric patients with achondroplasia with open epiphyses (growth plates). This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Previously, VOXZOGO was indicated for children who were 5 years of age and older. This expanded indication now includes children of all ages with open growth plates.

“We are pleased that VOXZOGO is now available for children of all ages with achondroplasia,” said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. “We are grateful for the collaboration of the achondroplasia community, physicians, and the children and their families who have played a crucial role in advancing this clinical program. We also look forward to further understanding the potential role of VOXZOGO in other genetic short stature conditions, including hypochondroplasia.”

“VOXZOGO is the only approved treatment for children with achondroplasia. Until now, it has only been approved in the U.S. for children aged 5 and older with open growth plates,” said William Wilcox, M.D., professor of human genetics at Emory University. “I am delighted that VOXZOGO is now approved for younger children where we hope to have potentially greater impact by starting treatment earlier and, as a result, a much longer treatment window.”

BioMarin conducted a randomized, double-blind, placebo-controlled Phase 2 clinical trial evaluating the safety and efficacy of VOXZOGO in children aged 5 and under (Study 111-206). Based on the results of this trial, together with evidence from the adequate and well controlled Phase 3 study in pediatric patients aged 5 years and older (Study 111-301), safety and effectiveness of VOXZOGO have been established in pediatric patients of all ages for the improvement in linear growth in children with achondroplasia with open epiphyses. The overall safety profile of VOXZOGO in children under 5 years of age was similar to that seen in older children.

Data from an open-label, long-term Phase 2 extension study was recently presented at the 2023 European Society for Paediatric Endocrinology Meeting in September. Over a four-year period, children aged 2 years and above who received VOXZOGO exhibited a mean (average) height Z-score improvement of 1.1 to 1.4 standard deviations (95% CI limits from 0.46 to 1.93) and a mean height gain of 6.3 to 7.8 centimeters (cm) (95% CI limits from 2.98 to 10.40 cm) when compared to untreated children with achondroplasia of the same age and sex. In addition, children under the age of 2 years, treated with VOXZOGO for three years, had a mean height Z-score improvement of 0.8 to 1.0 standard deviations (95% CI limits from 0.37 to 1.59) and a height gain between 3.5 and 3.9 cm (95% CI limits from 1.57 to 6.16 cm).

Since the introduction of VOXZOGO in 2021, the company has seen strong patient demand for the medicine worldwide. BioMarin has recently been able to secure increased fill-finish commitments in 2024 and beyond to meet this additional demand. There are approximately 800 children under 5 with achondroplasia in the U.S.

VOXZOGO is currently approved in Europe in children with achondroplasia who are 2 years of age and older with open growth plates. In September, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending marketing authorization to expand the indication for VOXZOGO for injection to treat children with achondroplasia aged 4 months and older whose epiphyses are not closed. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission later this year.  

VOXZOGO is also approved in Japan in children from birth who have achondroplasia with open growth plates. In addition, it is approved in Brazil in children who are 6 months and older with open growth plates as well as in Australia in children with achondroplasia who are 2 years of age and older with open growth plates.

Orphan Drug Designation in Hypochondroplasia

VOXZOGO also recently received orphan drug designation from the FDA for the treatment of hypochondroplasia, a genetic condition caused by a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene and characterized by impaired bone growth. While similar to achondroplasia, people with hypochondroplasia typically present with milder disproportionality and less severe short stature compared to achondroplasia.

BioMarin plans to initiate a pivotal development program in hypochondroplasia later this year.

About VOXZOGO (vosoritide) for Injection

In children with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in FGFR3. VOXZOGO, a C-type natriuretic peptide (CNP) analog, acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.

VOXZOGO is approved in the U.S. and indicated to increase linear growth in children with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trial(s). To fulfill this post-marketing requirement, BioMarin intends to use the ongoing open-label extension studies compared to available natural history.