New data from phase III HAVEN 6 study reinforce favourable safety and efficacy profile of Hemlibra in people with moderate or mild haemophilia A.

The data will be presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on 11 July 2022, in London, United Kingdom, and are planned to support a submission to the European Medicines Agency to update the label for Hemlibra to include non-severe haemophilia A patients.

In addition to HAVEN 6, data from the CHESS II (Cost of Haemophilia across Europe: a Socioeconomic Survey-II) and CHESS PAEDs studies will also be presented at ISTH 2022. These data show most adults with moderate or mild haemophilia A and more than half of children with moderate haemophilia A may not receive preventative treatments. This can result in worsened clinical burden, as more than 30% of adults and approximately 40% of children with moderate haemophilia A who were not taking preventative treatment in the study experienced at least three bleeds a year.

HAVEN 6 is a phase III, multicentre, open-label, single-arm study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild haemophilia A without factor VIII inhibitors. The primary analysis included data from 72 participants (69 men and three women) who warranted prophylaxis; 21 had mild haemophilia A without factor VIII inhibitors and 51 had moderate haemophilia A without factor VIII inhibitors at a median follow-up of 55.6 weeks. At baseline, 37 participants were receiving factor VIII prophylactic treatment and 24 had target joints.]The data show that Hemlibra maintained low treated bleed rates across the study period, 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). The results also show that Hemlibra’s safety profile was consistent with findings across various subpopulations of people with haemophilia A, from previous HAVEN and STASEY studies, with no new safety signals observed.

The most common adverse event (AE) related to treatment occurring in 10% or more people in the HAVEN 6 study was local injection site reactions (ISRs) (16.7%). Fifteen people (20.8%) reported a Hemlibra-related AE, of which the majority were local ISRs. One participant experienced a grade one thromboembolic event unrelated to Hemlibra. There were no deaths or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favourable safety profile.

Hemlibra is approved to treat people with haemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 95 countries worldwide, including the US and Japan for all severities of haemophilia A, and the EU for only severe haemophilia A. 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.