NHS England to fund broader use of Roche's haemophilia A therapy Hemlibra

NHS England said Wednesday that it will fund use of Roche's Hemlibra (emicizumab) for around 2000 people in the country with haemophilia A without factor VIII inhibitors. Simon Stevens, NHS chief executive, noted "giving patients access to world class, trailblazing drugs and therapies is a key part of the NHS Long Term Plan."

Hemlibra initially gained approval in Europe in 2018 for the treatment of bleeding episodes in patients with haemophilia A with factor VIII inhibitors, with the NHS deciding to fund use of the therapy in this indication later that year. Meanwhile, Hemlibra was cleared earlier in 2019 for routine prophylaxis of bleeding episodes in people with severe haemophilia A without factor VIII inhibitors.  

The latest approval was based on findings from two studies, with results from the HAVEN 3 trial showing that Hemlibra prophylaxis led to significant and clinically meaningful reductions in treated bleeds compared to no prophylaxis, and compared to prior treatment with factor VIII prophylaxis in a prospective intra-patient comparison. Meanwhile, in the HAVEN 4 study, Hemlibra showed a clinically meaningful control of bleeding when dosed every four weeks.

"This treatment has the potential to significantly improve the lives of people with haemophilia, especially children – reducing treatment time and even ending the dangerous bleeds which can lead to life-threatening cuts and life-changing damage," Stevens continued. The NHS noted that current treatments for haemophilia A involve multiple infusions every week, while Hemlibra is administered as a single injection once-a-week or fortnight.

Hemlibra generated sales of CHF 535 million ($545 million) in the first half of 2019. For related analysis, read ViewPoints: A closer look at why prescribers are embracing Roche's Hemlibra.

The news comes as Roche faces scrutiny over its deal to purchase Spark Therapeutics for $4.3 billion, with the UK Competition and Markets Authority, as well as US Federal Trade Commission, expressing concerns over the proposed tie-up. Some analysts have speculated that the delays are related to competitive concerns surrounding Hemlibra and a gene therapy for treating haemophilia A that is being developed by Spark Therapeutics (for additional analysis, see ViewPoints: Roche's haemophilia plans attract regulatory scrutiny).

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