Incyte's Jakafi OK'd By FDA For Blood Cancer PV

Delaware-based biopharmaceutical firm Incyte announced that the U.S. Food and Drug Administration (FDA) has approved a new indication for Jakafi (ruxolitinib) as treatment for patients with uncontrolled polycythemia vera (PV) who are intolerant to or have had an inadequate response to hydroxyurea.

Jakafi is a first-in-class JAK1/JAK2 inhibitor indicated for the treatment of intermediate or high-risk myelofibrosis. The drug works by targeting the overactive JAK pathway signaling, which is crucial for the development of both polycythemia vera and myelofibrosis. The drug is marketed by Incyte in the U.S. and elsewhere around the world by its partner Novartis.

Polycythemia vera is a myeloproliferative neoplasm (MPN) characterized by elevated hematocrit, which leads to a thickening of the blood as well as high counts of white blood cells and platelets. Excessive amounts of blood cells can cause the spleen to swell. This can also lead to bleeding problems and phlebitis or blood clots in the veins close to the skin surface. PV also puts patients at higher risk of stroke or heart attack. PV affects an estimated 100,000 in the U.S.

The FDA approved Jakafi based on the strength of results from the pivotal late-stage trial RESPONSE, which was conducted under a Special Protocol Assessment from the regulatory agency. Results show that Jakafi achieved greater hematocrit control and reduction in spleen volume against the comparator therapy. More patients under the Jakafi treatment arm also experienced complete hematologic remission with lower platelet and white blood cell counts. The FDA accepted Jakafi’s Supplemental New Drug Application (sNDA) under Priority Review last August.

“The approval of Jakafi for polycythemia vera underscores the importance of developing drugs matched to our increasing knowledge of the mechanisms of diseases. The trial used to evaluate Jakafi confirmed clinically meaningful reductions in spleen size and the need for phlebotomies to control the disease,” said Richard Pazdur, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

“The team at Incyte is proud that a second indication has been approved for Jakafi,” said Hervé Hoppenot, Incyte’s President and CEO.