Roche upbeat on European approval for 'Hemlibra'
Roche announced on Thursday that the European Commission has approved ‘Hemlibra’ (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A without factor VIII inhibitors.
The Swiss pharmaceuticals giant said Hemlibra could be used in all age groups, and could also now be used at multiple dosing options - once weekly, every two weeks, or every four weeks - for all indicated people with haemophilia A, including those with factor VIII inhibitors.
It said the approval was based on results from the pivotal HAVEN 3 and HAVEN 4 studies.
In the HAVEN 3 study in people with haemophilia A without factor VIII inhibitors, Hemlibra prophylaxis led to “statistically significant and clinically meaningful” reductions in treated bleeds compared to no prophylaxis, and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison.
Roche said that in the HAVEN 4 study in people with haemophilia A with and without factor VIII inhibitors, Hemlibra showed a “clinically meaningful” control of bleeding when dosed every four weeks.
“We are delighted that now people with severe haemophilia A without inhibitors in the EU will also have the opportunity to benefit from Hemlibra, which has been shown to significantly reduce bleeds compared to no prophylaxis and compared to prior factor VIII prophylaxis,” said Dr Elena Santagostino, director of the Hemophilia Unit at the Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre of the Cà Granda Foundation, Maggiore Hospital Policlinico in Milan.
“We are hopeful that the three different dosing options will allow people with haemophilia A and their physicians to choose the option that's right for them, based on their lifestyle and preferences.”
Sandra Horning, Roche’s chief medical officer, described the approval as a “landmark moment”, as Hemlibra was the first new class of treatment for people with severe haemophilia A without inhibitors in almost two decades.
“Moreover, Hemlibra can effectively control bleeds while offering subcutaneous dosing once weekly, every two weeks or every four weeks,” Horning said.
“We will continue to work with EU member states, to bring this important treatment to those in need as quickly as possible.”