The Food and Drug Administration has granted breakthrough therapy designation for ruxolitinib — Incyte Corporation’s treatment for patients with acute graft-versus-host disease.
The drug, trademarked Jakafi, is the first approved treatment available for patients with GVHD.
GVHD is an often fatal complication from stem cell transplants where the transplanted immune cells attack the patient’s body.The skin, digestive tract, and liver are the most commonly affected organs in patients with GVHD.
Breakthrough therapy designation is designed to expedite the development and review of drugs for serious or life-threatening conditions and to help ensure people have access to them through FDA approval as soon as possible.
The criteria for awarding breakthrough therapy designation require preliminary clinical evidence that demonstrates the drug may have clinically significant improvement over available therapy.
In March 2016, Incyte and Lilly agreed to amend their license, development and commercialization agreement to enable Incyte to independently develop and commercialize ruxolitinib for GVHD. In April 2016, Incyte and Novartis also agreed to amend their collaboration and license agreement, granting Novartis the rights to research, develop and commercialize ruxolitinib for GVHD outside the U.S.
Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. Food and Drug Administration, as Jakafi, for treatment of people with polycythemia vera who have had an inadequate response to or are intolerant of hydroxyurea medication. Jakafi is also indicated for treatment of people with intermediate or high-risk myelofibrosis.