CHMP adopts positive opinion for Roche’s prophylactic treatment, Hemlibra

The EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra (emicizumab) — Roche’s prophylactic treatment for people with haemophilia A with inhibitors.

Hemlibra is a bispecific factor IXa- and factor X-directed antibody, designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for people with haemophilia A. It can be administered by an injection or ready-to-use solution under the skin once a week.

Currently, the marketing authorisation application for this treatment is being reviewed under accelerated assessment. Based on this positive opinion, it is anticipated that a final decision on the approval of Hemlibra should be received from the European Commission soon.

“Many people with haemophilia A develop inhibitors, which greatly impacts their ability to treat or prevent bleeds and diminishes their quality of life,” said Brian O’Mahony, chief executive of the Irish Haemophilia Society and president of the European Haemophilia Consortium. “It’s been over 20 years since we last saw a new medicine for people with haemophilia A with inhibitors, so there is a pressing need for innovative treatments that control bleeding and decrease the negative impact on quality of life.”

“This positive CHMP opinion represents a significant step towards bringing this new treatment to people with haemophilia A with inhibitors in Europe,” said Dr Sandra Horning, Roche’s chief medical officer and head of Global Product Development. “Hemlibra has been shown to effectively reduce the frequency of bleeds compared to currently available medicines, and with once-weekly administration by injection under the skin, it could also greatly reduce the treatment administration burden, particularly for young children with haemophilia A with inhibitors and their families.”

This positive opinion has been based on the data of two clinical studies — HAVEN 1 and HAVEN 2. The CHMP has recommended Hemlibra be used for all age groups.