UK's NICE says no to Novartis' Jakavi for myelofibrosis in draft guidance

UK drugs watchdog the National Institute for Health and Clinical Excellence (NICE) has today (February 13) issued new draft guidance not recommending the use on the National Health Service of Swiss drug major Novartis’ (NOVN: VX) Jakavi (ruxolitinib) for the treatment of disease-related splenomegaly (enlarged spleen) or symptoms in adults with primary myelofibrosis, or myelofibrosis secondary to polycythemia vera or essential thrombocythemiai.

The independent Appraisal Committee considered that myelofibrosis symptoms (especially itch and fatigue) and spleen size are significant, and that improving these would be beneficial to the wellbeing of patients. The Committee concluded that ruxolitinib was effective in reducing spleen size and in treating symptoms, such as itch and fatigue. However, the Committee concluded that although it was plausible that ruxolitinib could offer a survival benefit, it noted uncertainties in the data used by the manufacturer to estimate the degree of survival benefit.

Novartis expressed its disappointment at the draft guidance for Jakavi. The draft guidance has also been met with dismay by patient groups and clinicians alike who only saw the drug made available in the UK last September. Jakavi is the first and only targeted treatment in this disease area and offers a “step change” in treating enlarged spleen and symptoms in patients with myelofibrosis, the company noted.