Hemophilia

Rebinyn Approved to Treat Hemophilia B

By June 01, 2017

Rebinyn, a recombinant DNA-derived coagulation Factor IX concentrate, temporarily replaces the missing coagulation Factor IX in hemophilia B patients.

Investigational Hemophilia B Gene Therapy Granted Breakthrough Status

By January 30, 2017

The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the investigational gene therapy, AMT-060 (uniQure), for the treatment of severe hemophilia B.

Hemophilia A Treatment Gains Expanded Approval

By December 27, 2016

Adynovate, an extended circulating half-life recombinant Factor VIII (rFVIII), was initially approved for use in patients aged ≥12 years with hemophilia A for on-demand treatment and control of bleeding episodes, and routine prophylaxis to reduce the frequency of bleeding episodes.

Emicizumab Prophylaxis Reduced Number of Bleeds in Hemophilia A Trial

By December 22, 2016

The primary endpoint was the number of bleeds over time among patients with emicizumab prophylaxis or no prophylaxis.