UK patients with a rare form of blood cancer will now, for the first time, have access to a targeted therapy against their disease following the launch of Novartis' Jakavi in the country.

Myelofibrosis is a rare, potentially life-threatening blood cancer characterised by bone marrow failure, enlarged spleen and debilitating symptoms including extreme fatigue, poor quality of life and weight loss, and shortened survival.

Jakavi (ruxolitinib) is a first-in-class JAK 1 and JAK 2 inhibitor, and is now available to treat patients in the UK with disease-related enlarged spleen or various forms of myelofibrosis, which affects around 0.34-0.76 people in every 100,000.

The drug received European approval late last month on the back of data from the COMFORT (COntrolled MyeloFibrosis Study with ORal JAK Inhibitor Therapy) clinical trail programme, which demonstrated that Jakavi was able to provide significant clinical benefit by decreasing spleen size, improving symptoms and impacting overall survival.

For example, findings of COMFORT-I study demonstrated that 41.9% of Jakavi-treated patients achieved at least a 35% reduction in spleen volume (equivalent to a cut in palpable spleen size by 50%) at 24 weeks, compared to 0.7% of patients in the placebo arm.

New standard of care?

None of the currently available treatments specifically target the underlying molecular defect that drives this disease, Novartis said, and the launch of Jakavi in the UK “has the potential to offer patients with myelofibrosis a new standard of care, for the first time," added Consultant Haematologist Claire Harrison, from Guy's and St. Thomas' NHS Foundation Trust in London. 

A company spokesperson told PharmaTimes UK News that Jakavi is available in three different dosages: 5mg, 15mg and 20mg, with price tags for a one-month's supply of £1,800, £3,600 and £3,600 (all exc VAT), respectively.

The question is, will it be endorsed by cost regulators for National Health Service use? 

Well, "with regards to an appraisal, we are expecting Jakavi to be appraised by NICE in the coming months", the spokesperson said, but added that "it is completely up to NICE when they choose to appraise drugs in a specific therapy area and so we cannot be certain of timing".