SAN RAFAEL, Caliph., August 19, 2020 / PRNewswire / – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogenic roxaparvovec gene therapy for severe hemophilia A. August 18, 2020. The FDA issues a CRL to indicate that the appraisal cycle for an application is complete and that the application is not in its current form for approval. “Data-reactid =” 14 “>SAN RAFAEL, Caliph., August 19, 2020 / PRNewswire / – BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) to the Company’s Biologics License Application (BLA) for valoctocogenic roxaparvovec gene therapy for severe hemophilia A. August 18, 2020. The FDA issues a CRL to indicate that the appraisal cycle for an application is complete and that the application is not in its current form for approval.
BioMarin Pharmaceutical logo (PRNewsfoto / BioMarin Pharmaceutical Inc.)
November 2019, and the last patient to follow up for two years in November 2021. “data-reactid =” 31 “> After previously agreeing with the Agency on the amount of data needed to support the BLA, the FDA introduced a new recommendation for two years of data from the company’s ongoing study 270-301 (Phase 3) to provide substantial evidence of a lasting effect using annual blood velocity (ABR) as the primary endpoint.The Agency first informed the company of this recommendation in the CRL, as they have not increased this at any time during development or review.The Agency recommended that the company complete the Phase 3 study and provide two-year data on safety and efficacy monitoring across all participants. ‘ The FDA concluded that the differences between Study 270-201 (Phase 1/2) and the Phase 3 study limited their ability to rely on the Phase 1/2 study to support sustainability of effect. 3-study was fully enrolled November 2019, and the last patient will complete two November follow-up in November 2021.
The company plans to meet with the Agency in the coming weeks to agree on the next steps to obtain approval.
“We continue to work with the hemophilia community and to lead the way to the first ever therapy in hemophilia A,” said Jean-Jacques Bienaimé, President and Chief Executive Officer of BioMarin. “We are surprised and disappointed that the FDA has for the first time introduced new expectations in the Complete Response Letter. We are confident in valoctocogenic roxaparvovec gene therapy and its potential to redefine the treatment paradigm for people with hemophilia A.”
The application was based on the Phase 3 study interim analysis of study participants treated with research product produced by the over-commercialized process and three-year data from the Phase 1/2 study. The FDA had granted valoctocogenic roxaparvovec Priority Review status and Breakthrough Therapy and Orphan Drug designations. Valoctocogenic roxaparvovec maintains Breakthrough Therapy and Orphan Drug indications.
The ongoing clinical trials of valoctocogenic roxaparvovec will continue, while BioMarin is investigating further steps to obtain approval. The European Medicines Agency (EMA) review of the Marketing Authorization (MAA) application for valoctocogen roxaparvovec is ongoing.
People living with hemophilia A deficient enough functioning Factor VIII protein to help their blood clot and are at risk for painful and / or potentially life-threatening bleeding from even modest injuries. In addition, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeding in their muscles or joints. Individuals with the most severe form of hemophilia A make up about 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much reduced tendency to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of Factor VIII replacement infusions, intravenously up to two to three times a week or 100 to 150 infusions per year. Despite this regimen, many people experience breakthrough bleeding, which leads to progressive and debilitating joint damage, which can have a major impact on their liveability.
Hemophilia A, also known as Factor VIII deficiency as classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed from parents to children, about 1/3 of the cases are caused by a spontaneous mutation, a new mutation that is not inherited. About 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 six commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit www.biomarin.com. Information on the BioMarin website is not included with reference in this press release.
June 30, 2020, and future applications and reports by BioMarin. BioMarin undertakes no obligation or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations. “Data-reactid =” 46 “> This press release contains forward-looking statements regarding the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements regarding: (i) the development of BioMarin’s Valoctocogene roxaparvovec program in general; (ii) further regulatory interactions with the FDA valoctocogenic roxaparvovec: (iii) possible requirements for valoctocogenic roxaparvovec approval, including the suitability of Study 270-301 to meet such requirements; (iv) conducting ongoing clinical trials for valoctocogenic roxaparvovec; ) and the ongoing review of the marketing authorization application for valoctocogenic roxaparvovec by the European Medicines Agency. of current and planned preclinical study and clinical trials of valoctocogenic roxaparvovec, including the possible one-year extension of Study 270-301; the content and timing of the expected follow-up meeting with the FDA; the content and timing of decisions by the FDA, the European Commission and other regulatory authorities; and those other risks that are detailed from time to time under the heading “Risk Factors” and elsewhere in BioMarin’s Securities and Exchange Commission (SEC) applications, including BioMarin’s Quarterly Report on Form 10-Q for the quarter June 30, 2020, and future applications and reports by BioMarin. BioMarin undertakes no obligation or obligation to update any forward-looking statements contained in this press release as a result of new information, future events or changes in its expectations.
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