European Commission approves label expansion of Hemlibra to include people with moderate haemophilia A in the EU
Roche announced that the European Commission approved the expansion of the Hemlibra (emicizumab) European Union (EU) marketing authorisation. The label will now include 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. Haemophilia A affects around 900,000 people worldwide, approximately 14% of whom have a moderate form of the disease
While the treatment and care of people with 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 an individual’s factor VIII levels, is not always reflective of bleeding behaviour. Many people with moderate haemophilia A may not be receiving prophylactic treatments and could have a worsened clinical burden. Approximately 85% of people with moderate haemophilia A have bleeds within a given year and one in three have long-term joint problems, many of which require surgery and impact quality of life.
This approval is based on the results of the phase III HAVEN 6 trial, in which 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.
HAVEN 6 is a phase III clinical study designed to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with non-severe haemophilia A without factor VIII inhibitors. Data from the primary analysis of HAVEN 6 was presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on 11 July 2022. The analysis included data from 72 participants, including three women, 51 (70.8%) of whom had moderate haemophilia A without factor VIII inhibitors. The data indicate that Hemlibra has effective bleed control and a favourable safety profile in people with non-severe haemophilia A without factor VIII inhibitors, with no new safety signals identified. Hemlibra achieved clinically meaningful bleed control, with 66.7% of participants experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment, and 88.9% experiencing no joint bleeds that required treatment. Model-based annualised bleed rates (ABR) remained low throughout the evaluation period at 0.9 (95% CI: 0.55-1.52).
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