The agency said Friday that it had approved Celgene’s Inrebic (fedratinib), the first new drug approved for the disease – a type of rare blood cancer – since the approval of Incyte’s Jakafi.
The Food and Drug Administration has approved the first new drug for a type of rare blood cancer in nearly a decade.
The FDA said Friday that it approved Celgene’s Inrebic (fedratinib) for intermediate-2 or high-risk primary or secondary myelofibrosis. Myelofibrosis is a type of bone marrow cancer in which scar tissue forms in the bone marrow, and blood cell production moves from there to the spleen and liver, causing enlargement of the organs, particular spleen enlargement, or splenomegaly. Primary myelofibrosis is when the disease happens on its own, while secondary myelofibrosis derives from precedent disorders like polycythemia vera and essential thrombocythemia, which respectively result from excess production of red blood cells and platelets.
Prior to Inrebic’s approval, the only drug approved for myelofibrosis was Incyte’sJakafi (ruxolitinib), which the FDA approved in 2011. Both drugs belong to a class known as JAK inhibitors. Jakafi is intermediate or high-risk myelofibrosis, including primary and secondary disease, along with polycythemia vera.
Shares of Celgene were up a little more than 1 percent on the Nasdaq Friday afternoon. The company agreed to be acquired by Bristol-Myers Squibb in January for $74 billion. Inrebic was touted as a key asset in the deal.
“Prior to today, there was one FDA-approved drug to treat patients with myelofibrosis, a rare bone marrow disorder,” FDA Office of Hematology and Oncology Products acting director Richard Pazdur said in a statement. “Our approval today provides another option for patients.”
The fact that the FDA has not approved a new drug for myelofibrosis since 2011 is not for lack of trying on the part of other companies.
Gilead Sciences sold rights to its JAK inhibitor, momelotinib, to Sierra Oncology in August 2018. According to results Gilead had announced in November 2016, although one Phase III study showed the drug was non-inferior to Jakafi, the other showed it did not beat physician’s choice of best available therapy among patients who had previously failed on Jakafi.
Meanwhile, CTI Biopharma has for several years developed another JAK inhibitor, pacritinib, but has run into problems. The FDA placed a full clinical hold on the drug in 2016 after patients experienced brain bleeds and heart attacks in one of its Phase III trials. The hold was lifted in January 2017, and the company now plans another Phase III study.