Full Press Release Details
SAN RAFAEL, Calif. , Dec. 23, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) announced today that the European Medicines Agency (EMA) validated the Company's Marketing Authorization Application (MAA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. The MAA review will commence in January 2020 under accelerated assessment.
Recognizing valoctocogene roxaparvovec for its potential to benefit patients with unmet medical needs, EMA granted access to its Priority Medicines (PRIME) regulatory initiative in 2017 and recently granted BioMarin's request for accelerated assessment of this MAA, potentially shortening the review period. This submission is based on an interim analysis of study participants treated in an ongoing Phase 3 study with material from the to-be-commercialized process and updated three-year Phase 1/2 data. This submission marks the first marketing application submission in Europe for a gene therapy product for any type of hemophilia.
The U.S. Food and Drug Administration (FDA) has granted valoctocogene roxaparvovec Breakthrough Therapy designation. Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the EMA.
"We are pleased that the agency has recognized the potential scientific advancement that valoctocogene roxaparvovec could bring to people with severe hemophilia A," said Hank Fuchs , M.D., President, Global Research and Development at BioMarin. "We continue to move thoughtfully and urgently through the regulatory review process to deliver a treatment that we believe has the potential to make a meaningful difference to people with hemophilia A. We also believe that the field of gene therapy offers the possibility of changing the practice of medicine in many rare genetic diseases like hemophilia A."
About Hemophilia A
People living with hemophilia A lack enough functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 43 percent of the hemophilia A population. The standard of care for such individuals with hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.
Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. The Company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com . Information on BioMarin's website is not incorporated by reference into this press release.
Forward Looking Statement
BioMarin® is a registered trademark of BioMarin Pharmaceutical Inc.
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SOURCE BioMarin Pharmaceutical Inc.