The FDA has agreed to allow Alnylam (NASDAQ: ALNY) and Sanofi (NYSE: SNY) to resume trials of their hemophilia therapy, fitusiran. Previously, these companies had to halt their fitusiran studies after a patient who had advanced into an extension study died because of a fatal blood clot. Is this news good for patients and investors?
Hemophilia A is caused by a deficiency of blood coagulation factor VIII and hemophilia B is caused by a deficiency of blood coagulation factor IX. In both cases, the low levels of clotting factor put patients at significant risk of bleeding and hemorrhages. It's estimated that there are about 150,000 patients with hemophilia A and 30,000 patients with hemophilia B, globally.
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Source: Fool.com