Full Press Release Details
Expanded Indication in the U.S. Now Includes Children of all ages with Achondroplasia
SAN RAFAEL, Calif. , Oct. 20, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN ), 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 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.
Patient Support Accessing VOXZOGO
To reach a BioMarin RareConnections ® Case Manager, please call, toll-free, 1-833-VOXZOGO (1-833-869-9646) or e-mail [email protected] . For more information about VOXZOGO, please visit www.voxzogo.com . For additional information regarding this product, please contact BioMarin Medical Information at [email protected] .
About Achondroplasia
Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature in humans, is characterized by slowing of endochondral ossification, which results in disproportionate short stature and disordered architecture in the long bones, spine, face, and base of the skull. This condition is caused by a change in the FGFR3 gene, a negative regulator of bone growth.
More than 80% of children with achondroplasia have parents of average stature and have the condition as the result of a spontaneous gene mutation. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being tested in children whose growth plates are still "open," typically those under 18 years of age. Approximately 25% of people with achondroplasia fall into this category.
VOXZOGO U.S. Important Safety Information
What is VOXZOGO used for?
What is the most important safety information about VOXZOGO?
What are the most common side effects of VOXZOGO?
How is VOXZOGO taken?
What should you tell the doctor before or during taking VOXZOGO?
You may report side effects to BioMarin at 1-866-906-6100. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch , or call 1-800-FDA-1088.
Please see additional safety information in the full Prescribing Information and Patient Information .
Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's unparalleled research and development capabilities have resulted in eight transformational commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit www.biomarin.com .
Forward-Looking Statements
BioMarin ® and VOXZOGO ® are registered trademarks of BioMarin Pharmaceutical Inc.
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