SAN RAFAEL, Calif. — BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced that positive early results from an investigator-sponsored Phase 2 study of VOXZOGO® (vosoritide) in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Toronto, Canada, March 12-16, 2024. Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate VOXZOGO’s positive impact on quality of life.
Results in Hypochondroplasia
Andrew Dauber, M.D., will present positive results from his investigator-sponsored Phase 2 study of VOXZOGO in children with hypochondroplasia. The annualized growth velocity (AGV) increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period (mean difference: 1.81 cm/year, p<0.0001). No new safety signals were reported.
“We are encouraged by preliminary results from the first study evaluating VOXZOGO in children with hypochondroplasia, suggesting the medicine has the potential to impact growth outcomes for this group of patients,” said Dr. Dauber, who is the lead study author and Chief of Endocrinology at Children’s National Hospital in Washington, D.C. “There are currently no approved medicines for children with hypochondroplasia, so we look forward to gathering additional data to deepen our understanding of VOXZOGO in hypochondroplasia and other growth-related conditions.”
In late 2023, BioMarin launched the pivotal clinical trial program studying the safety and efficacy of VOXZOGO in children with hypochondroplasia. A multinational observational study in children with hypochondroplasia (111-902) is currently recruiting participants, and the company plans to enter the treatment phase (Phase 3 trial) later this year.
New Data Highlights VOXZOGO Long-Term Efficacy, Safety and Quality of Life in Achondroplasia
7-year Phase 2 Results in Achondroplasia
Data from two studies of VOXZOGO in children ≥5 years of age with achondroplasia demonstrated a sustained improvement in AGV for each year that the participants were treated. In the Phase 2 study with more than 7 years of follow up, the mean increase in growth across each year of age up to 16 years compared with untreated participants was 1.63 cm/year for boys and 1.33 cm/year for girls. Separate cross-sectional comparative analysis assessing increase in height over a 7-year period showed an additional height gain of 11.03 cm with use of VOXZOGO compared to matched untreated children over the same period.
4-year Phase 3 Results in Achondroplasia
The same analyses were produced for the Phase 3 trial in which children with achondroplasia have a mean treatment follow-up of 4 years. The mean increase in growth across each year of age up to 17 years was 1.73 cm/year for boys and 1.46 cm/year for girls. A separate cross-sectional comparative analysis assessing increase in height over a 3-year period showed an additional height gain of 5.75 cm with use of VOXZOGO compared to matched untreated children over the same period.
3-year Phase 3 Quality of Life Results in Achondroplasia
Results from a separate analysis of the Phase 3 study showed that VOXZOGO improved health-related quality of life (HRQoL) among children with achondroplasia, particularly those associated with physical activities. After 3 years, the mean increase in Quality of Life in Short Stature Youth (QoLISSY) physical domain score was 6.0 as reported by caregivers and 6.3 as reported by children. These improvements were even more pronounced in children who grew more (for those with ≥ 1 SD increase in height z-score, the mean increase in physical domain score was 11.4 as reported by caregivers and 8.5 as reported by children).
“The clinical results at ACMG reinforce the long-term benefit we are seeing in children treated with VOXZOGO, with a meaningful median height gain of more than 11 cm in children with achondroplasia following 7 years of treatment,” said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. “We are particularly encouraged by new data demonstrating the potential impact height gains from VOXZOGO can have on quality of life, an outcome of incredible importance for children and families impacted by achondroplasia.”
“Achondroplasia can have a negative effect on a child’s quality of life, including physical, social and emotional aspects,” said Melita Irving, M.D., study investigator and consultant clinical geneticist at Guy’s and St. Thomas’ NHS Foundation Trust in London. “Our studies showed that increasing height with long-term administration of VOXZOGO can result in meaningful improvements in quality-of-life measures for children with achondroplasia.”
About VOXZOGO
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.
About BioMarin
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 the 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.
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