Expanding Hemlibra’s EU Approval for Moderate Hem A Recommended
Hemlibra is approved in more than 110 countries to address bleeding events
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Hemlibra’s (emicizumab) approval be expanded to include people with moderate hemophilia A without inhibitors.
“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 hemophilia A,” said Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, in a press release.
Hemophilia A is caused by a missing or defective factor VIII (FVIII), a protein essential for blood clotting. Normally, this protein binds to factor IX (FIX) and factor X (FX) to initiate blood clotting and stop bleeding.
The deficiency in FVIII can lead to uncontrolled and often spontaneous bleeds, especially in the joints and muscles, causing pain, long-term joint damage, and reduced mobility. Regardless of severity, the disease may significantly reduce the quality of life for patients and families.
Disease severity is determined by the amount of FVIII produced, but this doesn’t always reflect bleeding events. Around 14% of hemophilia A patients have moderate disease, but some have symptoms similar to severe disease.
“We know that people with moderate hemophilia A can still have bleeds that cause irreversible joint damage and impact quality of life,” Garraway said.
While treatments for severe hemophilia A are well established, those with moderate disease may not receive preventive treatments. These patients may have a worse clinical burden, with only 15% being without bleeds.
Hemlibra is approved in more than 110 countries to prevent or reduce the frequency of bleeding events with hemophilia A, with or without FVIII inhibitors, which are neutralizing antibodies that target FVIII, lowering standard replacement therapies’ effectiveness. It’s only approved for severe hemophilia A in the EU, however.
Hemlibra is an antibody that’s able to simultaneously bind to FIX and FX, mimicking FVIII activity and restoring the blood clotting process. It’s given as an under-the-skin injection once a week, every two weeks, or every four weeks (after initially being given once weekly for four weeks).
The CHMP recommendation to extend the use of Hemlibra to prevent bleeds in people with moderate hemophilia A (FVIII activity levels between 1% and 5%) without FVIII inhibitors and severe bleeding, is based on data from the Phase 3 HAVEN 6 trial (NCT04158648), as well as on real-world evidence.
The first analysis of the trial was based on data from 72 patients without FVIII inhibitors, most of whom (70.8%) had moderate hemophilia A. Before entering the study, participants had an average estimated rate of more than 10 bleeds a year.
Hemlibra was effective at controlling bleeds, with 66.7% of participants having no bleeds requiring treatment, 81.9% having no spontaneous bleeds requiring treatment, and 88.9% having no joint bleeds requiring treatment. The estimated annual bleeding rate was 2.3 bleeds overall, with 0.9 bleeds per year requiring treatment.
Hemlibra also had a positive safety profile, with no new safety concerns identified with moderate hemophilia A without FVIII inhibitors. The most common side effect reported in 16.7% of participants was reactions at the injection site.