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New Licence for Ruxolitinib

written by

Leukaemia Care, Charity

  • Pills 3

The European Medicines Agency (EMA) has approved a license application for ruxolitinib, for the treatment of patients with polycythaemia vera. Ruxolitinib is marketed by Novartis as Jakavi® and is also indicated for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis.

Polycythaemia vera (PV) is a rare condition, associated with a higher than normal production of red blood cells. The condition is most common in middle-aged and elderly people and is more common in men than women. Nearly 95% of people with PV have a mutation (or change) in a protein called JAK2 which is part of the mechanism regulating blood cell production, causing an overproduction of cancerous red blood cells. Ruxolitinib is a JAK1 and JAK 2 inhibitor which inhibits (or prevents) the excess production of these cells.

The outcome of the EMA decision is to licence ruxolitinib for the treatment of patients with polycythaemia vera who are resistant to or intolerant to treatment with hydroxyurea, which accounts for approximately 25% of patients with PV.

However, it is important to note that at present NHS patients in the UK are currently unable to access ruxolitinib in its new indication (for the treatment of polycythaemia vera) until it has been recommended for reimbursement by the relevant health technology appraisal body. The relevant body for England and Wales is the National Institute for Health and Care Excellence (NICE), for Scotland is the Scottish Medicines Consortium (SMC) and for Wales is the All Wales Medicines Strategy Group (AWMSG). The role of these appraisal bodies is to assess the cost-effectiveness of licenced drugs, in order to determine whether they will be reimbursed by the NHS.

NHS patients in Scotland can currently access ruxolitinib for the treatment of myelofibrosis, following its approval by the Scottish Medicines Consortium last week. Patients in England are also able to access ruxolitinib for the treatment of myelofibrosis via the Cancer Drugs Fund. However patients in Wales are currently unable to routinely access ruxolitinib following the decision by National Centre for Health and Care Excellence (NICE) not to recommend it.

If you are affected by this change and would like to speak to somebody about it, please call our CARE line on 08088 010 444.