FDA Grants Roche Breakthrough Therapy Designation on Hemophilia Drug

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The agency has granted breakthrough therapy designation to Roche’s hemophilia therapy for treating hemophilia A in patients without factor VIII inhibitors.

FDA has granted breakthrough therapy designation to Roche for Hemlibra (emicizumab-kxwh), a bispecific factor IXa- and factor X-directed antibody for treating patients with hemophilia A without factor VIII inhibitors, Roche announced on April 17, 2018. The designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates the medicines may demonstrate a substantial improvement over existing therapies.

“Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with hemophilia A without inhibitors, in an intra-patient comparison,” said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development, in a company press release. “We look forward to working with health authorities to make Hemlibra available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.”

FDA first granted Hemlibra breakthrough therapy designation in September 2015 and approved the drug in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with hemophilia A with factor VIII inhibitors. The drug was also recently approved by regulatory authorities in other countries, including by the European Commission in February 2018 for routine prophylaxis of bleeding episodes in patients with hemophilia A with factor VIII inhibitors.


Hemlibra was created by Chugai Pharmaceutical and is being co-developed by Chugai, Roche, and Genentech.

Source: Roche