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Novartis gets EU approval for myelofibrosis drug Jakavi

First alternative to bone marrow or stem cell transplant for rare condition

Novarits building

Novartis has been given the go-ahead in Europe to market Jakavi, its new drug licensed from Incyte Corp for symptoms associated with the rare bone marrow disease myelofibrosis.

The European Commission (EC) approved Jakavi (ruxolitinib) for the treatment of disease-related splenomegaly or symptoms in adult patients with various forms of myelofibrosis, a disorder in which healthy bone marrow is replaced by scar tissue.

The US FDA approved the twice-daily JAK 1 and JAK 2 inhibitor for the same indication in November 2011 under the Jakafi brand name, when the drug became the first alternative to a bone marrow or stem cell transplant - other than supportive care - for the disease.

Patients with the disease develop severe anaemia and other blood cell abnormalities, and blood forms in other organs such as the liver and spleen. Symptoms including fatigue, night sweats and pruritus, poor quality of life, weight loss and shortened survival. Patients typically live for six to 10 years, depending on the severity of the condition.

Jakavi is "an urgently needed new treatment option with the potential to make a real difference in patients' lives," according to Dr Claire Harrison, a consultant haematologist at Guy's Hospital in London, UK, and one of the investigators in the clinical trials programme for the drug.

"By targeting the dysregulated JAK pathway, Jakavi delivers a rapid and durable benefit that has the potential to become a new standard of care," she added.

EU approval was granted on the back of the COMFORT-I and COMFORT-II studies, which showed that more patients treated with ruxolitinib than placebo achieved reductions in spleen sizes of 35 per cent or more.

Early patient follow-up indicates a non-statistically significant trend towards improved survival, and Novartis is hopeful that this could become significant over time.

Analysts have suggested that Jakavi could follow a similar growth trajectory to Novartis' $4.7bn-a-year cancer drug Glivec (imatinib), which achieved blockbuster sales through the sequential addition of new indications and a fairly high price tag.

Myelofibrosis may represent a fairly modest market for Jakavi - which has been priced at around $80,000 per year in the US - but approval in other uses such as polycythemia vera, essential thrombocythemia and various solid tumours could help it make $1bn-plus at peak.

29th August 2012

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