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    Home > Medical News > Medical World News > The first hemophilia gene therapy will be approved by FDA in August

    The first hemophilia gene therapy will be approved by FDA in August

    • Last Update: 2020-02-21
    • Source: Internet
    • Author: User
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    Biomarin pharmaceutical announced Monday that the US FDA has accepted the company's application for permission to biologicals (BLA) for valoc tococogene roxaparvovec, a gene therapy for hemophilia A a The FDA also granted the application a priority review qualification and is expected to respond by August 21 this year If approved, it would be the first gene therapy approved in the United States to treat hemophilia In patients with hemophilia A, the gene encoding factor VIII is mutated, resulting in insufficient level of factor VIII For them, minor injuries can cause pain and life-threatening bleeding Patients with severe hemophilia usually have spontaneous bleeding of muscles and joints, which may cause permanent joint damage At present, the standard therapy for severe hemophilia A is prophylactic intravenous factor VIII Patients not only need to receive treatment 2-3 times a week, but even if they receive treatment, they may still have bleeding events, which has a significant impact on their quality of life Valoctocogene roxaparvovec is a transgenic gene therapy using AAV5 virus vector to deliver expression factor VIII Its advantage is that patients may only need to receive one treatment, and hepatocytes can continuously express factor VIII, thus eliminating the need for long-term prophylactic coagulation factor injection The treatment has obtained the breakthrough treatment certification granted by the US FDA, the prime Drug Certification granted by the EU, and the orphan drug qualification granted by EMA and FDA This application is based on the interim data analysis of the ongoing phase 3 clinical trials and the latest three-year efficacy data of the phase 1 / 2 clinical trials According to the latest data released at the international society of thrombosis and hemostasis 2019 (Isth 2019), patients who received gene therapy at a dose of 6e13vg / kg once in a 1 / 2 phase of clinical trial continued to control the annual blood loss rate (ABR) and the demand for using factor VIII in the third year after treatment During the three years of treatment, ABR and factor VIII utilization decreased by an average of 96% Patients no longer need injection of preventive factor VIII ▲ ABR data of hemophilia A patients receiving different doses of gene therapy (photo source: biomarin website) "The FDA's acceptance of this application and the granting of its priority review qualification is an important milestone for both the field of gene therapy and the hemophilia patient population," said Dr hank Fuchs, President of global research and development of biomarin "Decades of scientific research have made today's success possible We look forward to working with FDA to bring this breakthrough treatment to patients with hemophilia A " reference material: [1] BioMarin's Biologics License Application for Valoctocogene Roxaparvovec Accepted for Priority Review by FDA with Review Action Date of August 21, 2020 Retrieved February 20, 2020, from https://www.prnewswire.com/news-releases/biomarins-biologics-license-application-for-valoctocogene-roxaparvovec-accepted-for-priority-review-by-fda-with-review-action-date-of-august-21-2020-301008771.html Note: the purpose of this article is to introduce the progress of medical health research, not to recommend the treatment plan For guidance on treatment options, please visit a regular hospital Original title: express delivery of hemophilia gene therapy, which is expected to be approved by FDA in August A kind of
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