CHMP recommends expansion of EU label for Hemlibra to include people with moderate haemophilia A.- Roche

If approved, Hemlibra would also be indicated for the routine prophylaxis of bleeding episodes in people with haemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, who have moderate disease (FVIII greater than 1% and less than 5%) with a severe bleeding phenotype. It is estimated that people with moderate haemophilia A make up 14% of the haemophilia A population.

While the treatment and management of severe haemophilia A are well-established, there is less information and guidance on prophylaxis for moderate haemophilia A. Additionally, the severity of haemophilia A, traditionally measured by factor VIII levels, is not always reflective of bleeding behaviour. Some people with non-severe haemophilia may experience symptoms similar to those with severe haemophilia and would benefit from prophylaxis. All severities of haemophilia A may significantly reduce the quality of life for people affected, as well as their family and caregivers. Given that many people with moderate haemophilia A may not receive prophylactic treatments, they may endure a worsened clinical burden, with 85% having bleeds within their lifetime. This can lead to long-term joint problems, requiring interventions and impacting quality of life.

The CHMP recommendation is based on the results from the phase III HAVEN 6 study, as well as on real world data. The results from the study showed that Hemlibra demonstrated effective bleed control and a favourable safety profile in people with non-severe haemophilia A without factor VIII inhibitors, where prophylaxis was clinically indicated. A final decision regarding the approval of Hemlibra is expected from the European Commission in the near future. If approved, this update will provide an effective and convenient prophylactic treatment option with a favourable safety profile, for people in the EU with moderate haemophilia A with a severe bleeding phenotype.