Scotland's NHS to fund Roche's Hemlibra in haemophilia A

NHS patients in Scotland with haemophilia A are set to get access to Roche and Chugai’s prophylactic injection Hemlibra after officials said it was cost-effective.

NHS Scotland, which makes devolved decisions on whether to fund medicines, opted to commission the drug.

Hemlibra (emicizumab), an antibody that mimics the action of factor VIII, 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.

Jay Gardner, 22, a patient from Edinburgh, said he would be discussing the new treatment option with his doctor soon.

Although his condition is controlled with an extended half-life product, he has to take injections every three days to prevent bleeding events, and before that he was on treatment regimen involving injections every two days.

He said: “It’s definitely exciting. It’s life changing for haemophiliacs like myself, reducing the amount of injections needed.

“I am not on Hemlibra at the moment but I would like to try it. The style of injection, the fact it is subcutaneous and not intravenous allows the veins to rest.”

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.

People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. There is currently no cure for haemophilia A but prophylaxis with factor VIII has been recognised as the preferred treatment approach to help prevent and reduce the frequency of bleeds.

In a separate devolved funding decision the All Wales Medicines Strategy Group has recommended Biogen’s Fampyra (fampridine) to improve walking in adults with multiple sclerosis and walking disability.

Fampyra is recommended for use in all subtypes of MS, including relapsing remitting MS (RRMS), secondary progressive MS (SPMS), primary progressive MS (PPMS), and progressive relapsing MS (PRMS) that have either very limited or no treatment options, depending on disease severity.