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Rare bone marrow disease drug approved in US

FDA says Incyte Corp allowed to market Jakafi

Incyte Corp has been given the green light in the US to market Jakafi, the first drug specifically approved to treat the rare bone marrow disease myelofibrosis.

The US Food and Drug Administration (FDA) cleared Jakafi (ruxolitinib; formerly INC424) yesterday for patients with intermediate- or high-risk myelofibrosis, a disease in which the bone marrow is replaced by scar tissue resulting in blood cells being made in organs such as the liver and the spleen. Symptoms including fatigue, night sweats and pruritus, poor quality of life, weight loss and shortened survival.

In trials, Jakafi cut spleen sizes by 35 per cent compared to placebo, and increased the proportion of patients with improvements of 50 per cent or more in myelofibrosis symptoms.

The approval is also good news for Novartis, which has rights to ruxolitinib outside the US under the terms of a licensing deal signed with Incyte in 2009.

Jakafi is the first in a new class of compound which act as oral inhibitors of the enzymes JAK1 and JAK2, whose functioning is disrupted in myelofibrosis. They are believed to play a role in regulating blood and immunological function.

"Jakafi represents another example of an increasing trend in oncology where a detailed scientific understanding of the mechanisms of a disease allows a drug to be directed toward specific molecular pathways," said Richard Pazdur, director of the Office of Haematology and Oncology Products at the FDA.

While the number of patients suffering from myelofibrosis is small at around one case per 100,000 people,  JAK1 and JAK2 disruption is also implicated in various other indications, including another bone marrow disease known as polycythemia vera, as well as other blood disorders and certain solid tumours.

Novartis has gone on record saying it has high hopes for Jakafi, which observers believe could follow a similar path to the firm's cancer drug Glivec (imatinib). This was initially approved in 2001 in a relatively small indication (second line treatment of chronic myeloid leukaemia) but grew quickly to multibillion-dollar sales thanks to a slew of follow-on indications.

Incyte has the distinction of being the first company to bring a JAK inhibitor to market, although Pfizer is developing its own candidate, called tofacitinib, in phase III testing as a treatment for rheumatoid arthritis and psoriasis.

Analysts have suggested that sales of Jakafi in myelofibrosis will likely be modest at about $100m a year, but additional indications could drive peak sales up above the $1bn threshold.

17th November 2011

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