Full Press Release Details
SAN RAFAEL, Calif. , Nov. 21, 2019 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN ) announced today that the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its investigational gene therapy, valoctocogene roxaparvovec, for adults with severe hemophilia A. Subject to completion of EMA's validation check, BioMarin anticipates the start of the MAA review to commence in January 2020 under accelerated assessment. BioMarin will provide an update in January 2020.
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 for a gene therapy product for any type of hemophilia.
The company remains on track to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) by the end of the year. The FDA has granted valoctocogene roxaparvovec Breakthrough Therapy designation. Valoctocogene roxaparvovec has Orphan Drug designation from the FDA and the EMA.
"We are grateful to the study participants, who have made this progress possible in the span of approximately four years since the first participant was enrolled in the clinical program," said Hank Fuchs , M.D., President, Global Research and Development at BioMarin. "We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy represents the next wave of innovation and potentially could be a meaningful advancement for treating people with severe 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 severe hemophilia A often experience painful, spontaneous bleeds into their muscles or joints. Individuals with hemophilia A diagnosed as severe make up 43 percent of the hemophilia A population. The standard of care for severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week. 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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| BioMarin Pharmaceutical Inc. | BioMarin Pharmaceutical Inc. |
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SOURCE BioMarin Pharmaceutical Inc.