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.