BioMarin Pharmaceutical Inc. announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5.
| The Supplemental New Drug Application is Based on Positive Results from Global Randomized Phase 2 Study in Infants and Young Children FDA set PDUFA Target Action Date of October 21, 2023 SAN RAFAEL, Calif., March 7, 2023 /PRNewswire/ --BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, announced today that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental New Drug Application (sNDA) for VOXZOGO® (vosoritide) for injection to expand treatment in the United States to include children with achondroplasia under the age of 5. Achondroplasia is the most common form of disproportionate short stature.
The FDA has set a PDUFA target action date of October 21, 2023 for the sNDA. “We are pleased that the FDA has accepted our sNDA and are working closely with the agency to facilitate completion of the review in a timely manner,” said Hank Fuchs, M.D., president, Worldwide Research and Development at BioMarin. “There are currently no approved pharmacological treatments in the United States for children under 5 with achondroplasia and this approval could potentially extend access to all children with achondroplasia, whose growth plates are not closed.” The sNDA is supported by results from a Phase 2 randomized, double-blind, placebo-controlled clinical trial, which demonstrated similar safety and efficacy profiles in children under 5 years of age as compared to those ages 5 years and older. In January, the European Medicines Agency (EMA) validated BioMarin’s application for extension of indication for VOXZOGO to treat children with achondroplasia under the age of 2. Approval of the submissions would mean VOXZOGO could potentially be prescribed as early as birth for more than 1,000 additional children eligible for treatment for achondroplasia. VOXZOGO is the first FDA and EMA approved treatment for children with achondroplasia with open epiphyses (bone growth plates). 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. About Achondroplasia Achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature, 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 change in the gene. The worldwide incidence rate of achondroplasia is about one in 25,000 live births. VOXZOGO is being studied 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. Please see additional safety information in the full Prescribing Information and Patient Information. 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. For additional information, please visit www.biomarin.com. Forward-Looking Statements 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; potential approvals in the U.S. and EU of BioMarin’s supplemental marketing applications for VOXZOGO and the number of additional children that will be eligible for VOXZOGO if such age expansions are accepted; and the timing of the PDUFA target action date for BioMarin’s sNDA 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.
SOURCE BioMarin Pharmaceutical Inc. | ||||||||||||
Company Codes: NASDAQ-NMS:BMRN |
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