March 14, 2019

European Commission approves Roche’s Hemlibra for people with severe haemophilia A

The European Commission has approved emicizumab (Hemlibra) as prophylaxis in people with severe haemophilia A without factor VIII inhibitors. The decision endorses a positive opinion from the Agency’s Committee for Medicinal Products for Human Use on January 31st.

Emicizumab was approved for prophylaxis in people with inhibitors in February 2018 and was approved for NHS use in July. The licence extension was granted in light of the results of the HAVEN 3 trial,1in which 152 patients aged over 12 were randomised to treatment with emicizumab 1.5 mg/kg or 3.0 mg/kg every 2 weeks (if they had been using on-demand factor VIII ), 1.5 mg/kg (if they had been using prophylaxis) or placebo.

After up to 24 weeks’ use, the annualised bleeding rate in placebo recipients was 38.2. Among individuals previously using on-demand treatment, this was reduced by 96% – 97% to 1.5 and 1.3; in this group, 56% of people using the lower dose and 60% of those using the higher dose had no treated bleeds. In individuals who had previously used prophylaxis, the annualised bleeding rate was 68% lower than it had been with prophylaxis. The most frequent adverse events associated with emicizumab are injection site reactions (19%), headache (15%) and arthralgia (10%).

For the NHS, the question whether to fund emicizumab for people without inhibitors is distinct from the issue of its use by patients with inhibitors, for whom other treatment options were limited, inconvenient and very expensive. It is therefore likely that a new appraisal will be carried out to determine the cost effectiveness of emicizumab relative to prophylaxis with conventional and extended half-life factor VIII.


  1. N Engl J Med 2018; 379:811-822

Roche Media Release