NHS Scotland has approved the reimbursement of Hemlibra (emicizumab), Roche’s severe congenital haemophilia A treatment for patients without factor VIII inhibitors.

The drug, which is developed by Roche’s Chugai Pharma subdivision, can be self-administered by injection under the skin at multiple dosing options (once weekly, every two weeks, or every four weeks). In clinical trials, Hemlibra dosed once-weekly or every two weeks led to a statistically significant and clinically meaningful reduction in treated bleeds compared to on-demand treatment.

Further, in pooled data a high proportion of patients with Haemophilia A who were treated, both with and without inhibitors, achieved zero treated bleeds. This continued to increase over time, from 70.8% in week 1 to 24 to 88.6% in weeks 73-96, the company revealed.

It is “vital” that people with haemophilia A in Scotland have access to the “latest, innovative treatments,” explained Dan Farthing-Sykes, chief executive officer of Haemophilia Scotland.

He went on to say that the news is “very welcome,” before saying that “compared to currently available treatment options which have to be infused directly into a vein, Hemlibra is injected just under the skin. It can also be given less frequently. This could have real impact on the quality of life of patients and their families. Our hope is that it will lead to fewer bleeds and therefore help patients successfully treat their condition in the long-term.”

Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, in severe cases leading to uncontrolled and often spontaneous bleeding. There is currently no cure for the disorder, but prophylaxis with factor VIII has been recognised as the preferred treatment approach to help prevent and reduce the frequency of bleeds.