Roche’s emicizumab in being included in the UK’s Early Access to Medicines Scheme for routine prophylaxis of bleeding episodes in patients with haemophilia A with factor VIII inhibitors.
The drug is a bispecific monoclonal antibody, designed to bring together proteins required to activate the natural coagulation cascade and restore the blood clotting process for haemophilia A patients. The therapy is used prophylactically, and can be administered subcutaneously.
The Medicines and Healthcare Regulatory Agency’s scientific opinion - awarded in the second stage of the EAMS eligibility process - is based largely on data from the Phase III HAVEN programme.
This shows that prophylactic use of emicizumab in inhibitor patients indicates a reduction of treated bleeds of 87 percent after 24 weeks compared to standard of care of ‘on-demand’ use of bypassing agents.
According to the MHRA, the overall safety profile of emicizumab also appears to be tolerable, with injection site reaction the most commonly reported adverse drug reaction, occurring in 19 percent of patients.
The main safety concern is around the development of thromboembolic events or thromboembolic microangiopathy, but this was considered to be related to concomitant use of bypassing agents, particularly cumulative doses of activated prothrombin complex concentrate, it noted.
The European Medicines Agency is currently reviewing Roche’s marketing application for the drug under an accelerated assessment. In the US it is also approved under the trade name Hemlibra.