European Commission Approves Treatment for Hemophilia A with Inhibitors

ČSH

Roche announced that the European Commission has approved the medicinal product Hemlibra for prophylaxis of bleeding episodes in people with hemophilia A with inhibitors. Approximately one in three people with severe hemophilia A develops antibodies against Factor VIII, causing replacement therapy to lose its effectiveness. This leads to a greater risk of life-threatening bleeds or recurrent bleeding episodes. People with hemophilia A and inhibitors face a 70% higher risk of death compared to those without inhibitors.

"We are pleased that the European Commission has approved the medicinal product and provided the first new drug in over twenty years for the treatment of hemophilia A with inhibitors," said Sandra Horning, Chief Medical Officer of Roche. "We are committed to working with EU member states to enable the fastest possible access to this important medication."

The approval is based on results from two large-scale clinical trials in which Hemlibra demonstrated superior efficacy compared to prior treatment with bypassing agents (BPA).

In the HAVEN 1 study in adults and adolescents (aged 12 and over), prophylactic administration of Hemlibra resulted in a statistically significant 87% reduction in treated bleeds.
Interim results from the HAVEN 2 study in children under 12 showed that 87% of children who received prophylactic Hemlibra experienced no treated bleeds.

The most common adverse effects, occurring in 10% or more of people treated with Hemlibra, included injection site reactions and headache. In three cases, thrombotic microangiopathy (TMA) occurred, and in two cases, a serious thrombotic event, when a cumulative average of more than 100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) (FEIBA) was administered over 24 hours or more during prophylactic Hemlibra treatment. The process demonstrated that the product significantly reduces bleeding even with longer follow-up.

Hemlibra represents a prophylactic treatment that can be administered as a subcutaneous injection once a week.