The European Commission has licensed Roche/Chugai’s Hemlibra for the routine prevention of bleeding episodes in patients with haemophilia A with factor VIII inhibitors.
Nearly one in three people with severe haemophilia A develop inhibitors, or resistance, to factor VIII replacement therapy, which can reduce its efficacy.
The presence of an inhibitor makes treating bleeds more difficult, as standard treatment does is ineffective and alternatives are “time-consuming and often burdensome to administer, especially for children, and can provide variable bleeding control,” the firm’s note.
The approval of Hemlibra (emicizumab) - a bispecific monoclonal antibody designed to trigger the natural coagulation cascade and restore the blood clotting process - provides a new treatment option where the standard of care is currently a bypassing agent (BPA) requiring infusion directly into the vein via a drip, either on demand or as prophylaxis to control bleeding.
Data from the data from the Phase III HAVEN programme showed that Hemlibra - which is dosed as a regular, once–weekly injection under the skin - reduced the number treated bleeds by 87 percent after 24 weeks compared to standard of care of ‘on-demand’ use of BPAs.
“Inhibitor development is currently the most challenging treatment complication seen with haemophilia A and is associated with considerable morbidity and a decreased quality of life,” noted Dr Gerry Dolan, consultant haematologist and centre director at Guys and St. Thomas' NHS Trust, and UK principal investigator in the HAVEN 1 study.
Hemlibra’s authorisation “offers a major advance in therapy for patients with inhibitors. This new, preventative, treatment has been shown to reduce the number of bleeding episodes compared to bypassing agents, which can significantly improve the physical well-being of patients,” he added.
The drug has already been available to patients in the UK via the country’s Early Access to Medicines Scheme, after regulators permitted its inclusion in the scheme last month.