New Analyses from Phase 2 and Phase 3 Extension Studies Continue to Demonstrate Sustained Improvement in Growth with no Change in Safety Profile
SAN RAFAEL, Calif., March 14, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will present updated data demonstrating the long-term benefit of treatment with VOXZOGO® (vosoritide) and new observational data on disease burden in children with achondroplasia. These data will be shared this week in an oral presentation and five posters at the 2023 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Salt Lake City, Utah.
The data will include seven-year results from the open-label, Phase 2 extension study in children with achondroplasia, providing further evidence that improvement in growth velocity with VOXZOGO is sustained as children grow older. Mean age- and sex-specific Annualized Growth Velocities (AGVs) in children treated with VOXZOGO were greater than corresponding mean AGVs in untreated children of the same age and sex and at all ages. When looking across all ages for the entire seven-year period, the mean (SD) difference in AGV versus untreated children was 1.90 (0.51) cm/year for boys and 1.36 (0.63) cm/year for girls in this analysis. A trend toward improvement in body proportion (upper:lower body segment ratio) over time continued to be apparent as children grew older.
Results from up to 3.5 years of the open-label extension of BioMarin's Phase 3 pivotal study of VOXZOGO also will be presented. Earlier data from this study supported the drug's approval in the United States, Europe, Brazil, Australia and Japan. In children on VOXZOGO, the mean (standard deviation [SD]) AGV improved from 4.26 (1.54) cm/year at baseline to 5.57 (0.76) cm/year after three years of treatment.
In both studies VOXZOGO remained well tolerated with no change in its adverse event profile.
"We are encouraged to see that as children remain on treatment, the growth promoting effect continues. We are observing sustained increases in growth velocity year on year," said Dr. Melita Irving, MBBS, consultant clinical geneticist at Guy's and St. Thomas' NHS Foundation Trust in London. "The trend toward improving proportionality also has the potential to have an impact on the lives of children with achondroplasia and we look forward to further follow up to better understand that data as they evolve."
"We are grateful to the children and families who continue to participate in the VOXZOGO achondroplasia clinical trials, as we gather longer term data and work toward our goal of providing this therapeutic option to children of all ages," said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin.
VOXZOGO addresses the root cause of achondroplasia, the most common form of disproportionate short stature. It is the first U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved treatment for children with achondroplasia with open epiphyses (bone growth plates). In January, the EMA validated BioMarin's application for extension of indication for VOXZOGO to treat children with achondroplasia under the age of 2 and earlier this month, the FDA accepted the company's Supplemental New Drug Application for children under 5, setting a PDUFA target action date of October 21, 2023.
BioMarin's presentations at ACMG include:
A Randomized Controlled Trial of Vosoritide in Infants and Toddlers with Achondroplasia
Abstract No. O22
Persistent Growth-Promoting Effects of Vosoritide in Children with Achondroplasia for up to 3.5 years: Update from Phase 3 Extension Study
Abstract No. P193
Health-Related Quality of Life (HRQoL) in Achondroplasia: Findings from LISA (Life Impact Study on Achondroplasia), a Multinational and Observational Study in Latin America
Abstract No. P233
Vosoritide Therapy in Patients with Achondroplasia: Early Experience and Practical Considerations for Clinical Practice
Abstract No. P339
Persistence of Growth Promoting Effects in Children with Achondroplasia Over Seven Years: Update from Phase 2 Extension Study with Vosoritide
Abstract No. P194
Objectives and Design of the Acorn Study: A Non-Interventional Study Evaluating Long-term Safety in Achondroplasia Patients Treated with Vosoritide
Abstract No. P338
About VOXZOGO® (vosoritide) for Injection
In patients 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 fibroblast growth factor receptor 3 gene (FGFR3). VOXZOGO, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Through BioMarin's broad clinical development program, the company has enrolled 250 children with achondroplasia from eight countries in seven clinical studies evaluating the safety and efficacy of VOXZOGO.
VOXZOGO is approved in the U.S. and indicated to increase linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication 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.
VOXZOGO is also approved in the EU, Brazil, and Australia in children with achondroplasia who are 2 years of age and older with open growth plates. It is also approved in Japan in children from birth who have achondroplasia with open growth plates.
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 fibroblast growth factor receptor 3 gene (FGFR3), 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?
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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.
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. For additional information, please visit www.biomarin.com.
This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: the development of BioMarin's VOXZOGO® (vosoritide) program generally; the potential benefits of VOXZOGO for children with achondroplasia, including the duration of such benefits and potential improvement in proportionality; the continued clinical development of VOXZOGO; and potential approvals in the U.S. and EU of BioMarin's supplemental marketing applications for VOXZOGO. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of VOXZOGO; any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Annual Report on Form 10-K for the quarter ended December 31, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.
BioMarin® and VOXZOGO® are registered trademarks of BioMarin Pharmaceutical Inc.
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