Novartis' ruxolitinab to treat rare blood cancer recommended for approval by NICE

By Liza Laws

- Last updated on GMT

© Getty Images
© Getty Images

Related tags blood cancer drug blood cancers NICE Uk Wales Novartis

Patients with a rare and incurable blood cancer, associated with an overproduction of blood cells, will soon have access to a small molecule Novartis treatment.

The medicines company will make Jakavi (ruxolitinab) – a JAK 1/2 inhibitor authorized to treat polycythaemia vera (PV) available in England and Wales.

The news comes as the National Institute for Health and Care Excellence (NICE) published its Final Draft Guidance (FDG) recommending ruxolitinib for eligible adults in England and Wales with PV that are resistant to or intolerant of the chemotherapy treatment hydroxycarbamide/hydroxyurea​ (HC/HU).

Blood withdrawal or venesection or HC/HU are the common treatment approaches for PV but approximately 24% of PV patients treated with the chemotherapy option will deliver resistance or intolerance resulting in inadequate disease control and an increased risk of progression, Novartis found in its research.

“There is a significant unmet need for people with polycythaemia vera in England and Wales, who live with a large symptom burden as a result of their condition,’’ said Claire Harrison, consultant haematologist, Guy's and St Thomas' NHS Foundation Trust, London.

“Today’s decision is a step in the right direction for providing additional treatment options that reduce the burden of these symptoms and improve disease progression, in this under-represented patient population.”

Research has shown in PV, the overproduction of red blood cells causes the blood to thicken which can cause stroke or heart attack. Other symptoms include headaches, fatigue, weakness, dizziness, or itchy skin.

Uncontrolled PV usually involves haematocrit levels (proportion of red blood cells in the blood) greater than 45%, elevated white blood cell count and/or platelet count which may be accompanied by debilitating symptoms and/or an enlarged spleen.

Findings from the Novartis international MPN Landmark survey, of patients with rare blood cancers including PV, demonstrated that 72% of PV patients who experienced symptoms suffered a reduction in quality of life. In addition to physical symptoms, approximately one-third of PV patients in the study felt anxious or worried about their disease.

Jon Mathias, co-chair of MPN Voice, a charity that supports and advocates on behalf of PV patients, said: “We welcome this recommendation from NICE, as polycythaemia vera can be an extremely debilitating illness that has a significant impact on patients’ lives in terms of day-to-day symptoms.

“It affects not only patients but also their families and carers and turns many everyday tasks into major hurdles. Ruxolitinib addresses a significant unmet need in patients who cannot tolerate or no longer respond to HC/HU.”

Novartis said its research showed an estimated 1,130 people are newly diagnosed with PV in the UK every year and that the disease is driven by the dysregulation of the JAK-STAT pathway. It is typically characterised by elevated haematocrit, the volume percentage of red blood cells in whole blood, which can lead to a thickening of the blood and an increased risk of blood clots, as well as an elevated white blood cell and platelet count.

This can cause serious cardiovascular complications, such as stroke and heart attack, resulting in increased morbidity and mortality. Additionally, patients with PV may have an enlarged spleen and symptoms that are frequent and burdensome, with an overall impact on quality of life similar to that seen with myelofibrosis.

“Today’s NICE recommendation in PV is an example of how we are reimagining medicine to transform the treatment of people with various blood cancers. The availability of ruxolitinib for eligible patients with PV in England and Wales will give them and their healthcare professionals more options in the management of this debilitating condition,” said Marie-Andree Gamache, president and managing director of Novartis UK and Ireland.


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