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BioMarin’s hemophilia gene therapy recommended for approval in Europe

June 24, 2022

The regulatory agency that recommends whether new drugs should be approved in Europe has thrown its support behind a gene therapy for the rare bleeding disorder hemophilia A, putting the potentially curative treatment one step closer to market.

People with hemophilia A have genetic mutations which impair their ability to make a blood-clotting protein called Factor VIII. They are therefore more likely to experience prolonged or even life-threatening bleeds. Most of the drugs currently used to treat hemophilia A contain engineered versions of Factor VIII, and are administered two to three times a week.

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