The European Commission approved the prophylactic use of Hemlibra in patients of all ages who do not have factor VIII inhibitors, announced the treatment’s manufacturer, Roche.
Hemlibra is a man-made antibody that targets both factor IXa and factor X clotting factors at the same time. The treatment is designed to bring these molecules together and facilitate the activation of the natural coagulation pathways, subsequently restoring the blood clotting process in people with hemophilia A.
It is now the only available prophylactic therapy that can be administrated to both patients with and without factor VIII inhibitors through subcutaneous (under the skin) injection and multiple dosing regimens — once weekly, or every two or four weeks.
“We are delighted that now people with severe hemophilia A without inhibitors in the E.U. 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,” Elena Santagostino, MD, PhD, a director at the Maggiore Hospital Policlinico of Milan, said in a press release.
“We are hopeful that the three different dosing options will allow people with hemophilia A and their physicians to choose the option that’s right for them, based on their lifestyle and preferences,” she added.
The E.U. regulatory agency’s decision was supported by positive clinical data from the HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) Phase 3 studies demonstrating Hemlibra’s potential to manage bleeds in hemophilia A patients.
The HAVEN 3 trial evaluated the therapy in 152 adults and adolescents with hemophilia A without factor VIII inhibitors. The study showed that preventive treatment, administrated once a week or every two weeks, could reduce all bleeding episodes by 96–97%, compared with no prophylactic treatment. Also, treatment with Hemlibra was found to reduce treated bleeds by 68% compared with previous prophylactic treatment, based on intra-patient comparisons.
In the HAVEN 4 trial, which enrolled 48 adult and adolescent patients with hemophilia A regardless of factor VIII inhibitors status, researchers evaluated the potential of Hemlibra to manage bleeds when dosed every four weeks. Results showed that after at least 24 weeks of treatment with Hemlibra, 56.1% of patients had zero bleeds, and 90.2% had three or fewer bleeds. These results confirmed that the treatment can effectively control bleeding, even when administrated every four weeks.
Pooled data from the HAVEN studies showed that the most common side effects associated with Hemlibra treatment were injection site reactions, joint pain, and headaches.
“Today’s approval is a landmark moment as Hemlibra is the first new class of treatment for people with severe hemophilia A without inhibitors in nearly 20 years,” Sandra Horning, MD, Roche’s chief medical officer and head of global product development, said.
Hemlibra is currently available in more than 60 countries worldwide to prevent or reduce the frequency of bleeds in hemophilia A patients with factor VIII inhibitors.