CHMP recommends expansion of EU label for Roche Hemlibra to include people with moderate haemophilia A
Hemlibra is approved as a treatment for people with haemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 100 countries worldwide.
Basel: Roche has announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended expansion of the Hemlibra (emicizumab) European Union (EU) marketing authorisation. 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 ≥1% and ≤ 5%) with a severe bleeding phenotype. It is estimated that people with moderate haemophilia A make up 14% of the haemophilia A population.
"We know that people with moderate haemophilia A can still have bleeds that cause irreversible joint damage and impact quality of life," said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product Development. "We're very pleased that the CHMP's recommendation brings us closer to potentially transforming the day-to-day lives of people in the EU living with moderate haemophilia A."
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.
Hemlibra is approved as a treatment for people with haemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 100 countries worldwide. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight phase III studies.
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