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Jakafi cleared for bone marrow disorder by FDA

Approval of Incyte drug could achieve it blockbuster status

Incyte Novartis Jakafi ruxolitinib 

Incyte's Jakafi has been approved in the US for a haematological disorder that could help propel the drug to blockbuster status.

The green light from the FDA is for polycythemia vera, a disease which affects around 100,000 people in the US that occurs when too many red blood cells are made in the bone marrow leading to bleeding problems, blood clots and potentially a stroke or heart attack.

Jakafi (ruxolitinib) is already approved to treat myelofibrosis, with Incyte recording $250m in US sales for the drug in the first nine months of 2014 and ex-US marketing partner Novartis booking $195m from the product in the same period. Analysts said earlier this year that the addition of polycythemia vera to the drug's label will drive sales above $1bn a year.

The JAK 1/2 inhibitor is the first therapy to be specifically approved to treat the bone marrow disorder, which is currently managed using another blood-cell depleting agent called hydroxyurea and phlebotomy, a procedure used to remove excess blood from the body.

Jakafi is approved for use in patients who have an inadequate response to or cannot tolerate hydroxyurea, said the FDA. Patients with polycythemia vera who fail to consistently maintain appropriate blood count levels have an approximately four times higher risk of suffering a major blood clot.

Richard Pazdur, director of the Office of Hematology and Oncology Products at the FDA, said the new indication for Jakafi "underscores the importance of developing drugs matched to our increasing knowledge of the mechanisms of diseases."

In the RESPONSE trial, 21% of patients treated with Jakafi showed a reduction in the need for phlebotomy and reduced spleen volume after 32 weeks' therapy, compared to 1% of patients on best-available therapy.

Incyte is also testing ruxolitinib in a series of other indications, including pancreatic cancer (phase III) and non-small cell lung, breast and colorectal cancers (phase II). The pancreatic cancer trial is comparing the drug to placebo on top of capecitabine therapy as a second-line therapy and is due to report results in 2016.

Other JAK inhibitors coming through the pipeline include Gilead Sciences' CYT387, acquired as part of its $510m takeover of YM Biosciences at the end of 2012 and Cell Therapeutics/Baxter's pacritinib. Sanofi was forced to discontinue development of its fedratinib (SAR302503) candidate after cases of a rare form of encephalopathy were seen in late-stage trials.

Article by
Phil Taylor

5th December 2014

From: Sales



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