NHS Scotland gives green light to funding haemophilia A drug Hemlibra

pharmafile | December 18, 2019 | News story | Research and Development NHS, haemophilia, haemophilia B, haemophilia a, nhs scotland 

NHS Scotland has commissioned Hemlibra (emicizumab), by Chungai Pharma UK Ltd and Roche Products Ltd, to treat people with congenital haemophilia A without factor VIII inhibitors.

Hemlibra is self-administered by injection, and has multiple dosing options ranging from once a week to once every four weeks.

In clinical trials, Hemlibra dosed once or every two weeks led to a statistically significant and clinically meaningful reduction in treated bleeds compared to on-demand treatment.

In trial data, Hemilibra treatment in patients with haemophila A, both with and without inhibitors, resulted in a high proportion achieving zero treated bleeds. This increased over time, from 70.8% in weeks 1-24 to 88.6% in weeks 73-96.

Dan Farthing-Sykes, CEO of Haemophilia Scotland, said: “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.”   

Richard Eaton, Rare Conditions Lead at Roche Products UK, said, “Roche and Chugai are very pleased to announce that Hemlibra will be available to patients in Scotland living with severe haemophilia A without factor VIII inhibitors. Hemlibra is the only prophylactic medicine that can be administered subcutaneously and maintains a sustained therapeutic level between doses.”

Haemophilia A is an inherited disorder in which a person’s blood does not clot properly, in severe cases it leads 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 treatment is aimed reducing the frequency of bleeds.

Conor Kavanagh

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