Regulatory Affairs

Roche’s Hemlibra secures FDA approval for hemophilia A with inhibitors

PBR Staff Writer Published 17 November 2017

Roche’s Hemlibra (emicizumab-kxwh) has been approved by the US Food and Drug Administration (FDA) for routine prophylaxis to prevent or reduce frequency of bleeding episodes in patients with hemophilia A who have factor VIII inhibitors.

Hemlibra is a bispecific factor IXa- and factor X-directed antibody. The therapy which has been approved for both adults and children, functions by bridging other factors in the blood to restore blood clotting in the affected patients.

The preventative (prophylactic) treatment is to be given weekly through an injection subcutaneously.

Roche chief medical officer and global product development head Sandra Horning said Hemlibra’s approval reflects a major advancement for people with haemophilia A with inhibitors, who have found it difficult to manage their bleeding disorder and haven’t had a new medicine for close to 20 years.

Horning added: “We believe Hemlibra will improve protection against bleeds and reduce the treatment administration burden for people with haemophilia A with inhibitors, and we are committed to helping them access this medicine.”

Hemlibra demonstrated its ability to reduce bleeds substantially in adults and children in two phase 3 clinical studies HAVEN 1 and HAVEN 2 for people with haemophilia A with inhibitors.

FDA Oncology Center of Excellence director Richard Pazdur said: “Reducing the frequency or preventing bleeding episodes is an important part of disease management for patients with hemophilia.

“Today’s approval provides a new preventative treatment that has been shown to significantly reduce the number of bleeding episodes in patients with hemophilia A with Factor VIII inhibitors.

“In addition, patients treated with Hemlibra reported an improvement in their physical functioning.”

Pazdur is also the acting director of FDA Center for Drug Evaluation and Research Office of Hematology and Oncology Products.

FDA has stated that Hemlibra comes with a warning that severe blood clots have been observed in patients who were also given a rescue treatment for their bleeds for 24 hours or more while taking the haemophilia A drug.

Hemlibra was developed by Genentech which is a member of the Roche Group.

Image: Genentech’s Hemlibra injection for treating bleeding in certain hemophilia A patients. Photo: courtesy of Business Wire.