Earlier this week, the FDA granted
priority review to a new drug application (NDA) for fedratinib for the treatment of myelofibrosis. The drug had previously been awarded an orphan drug designation for the treatment of primary and secondary myelofibrosis.
“The acceptance of the NDA and granting of priority review for fedratinib represent the first potential new treatment option after many years for patients affected by myelofibrosis,” said Jay Backstrom, MD, chief medical officer for Celgene, the developer of fedratinib, in a statement.
Fedratinib is an oral kinase inhibitor that acts against wild type and mutationally activated Janus associated kinase 2 (JAK2).
Celgene submitted the NDA based on results from a randomized, placebo-controlled phase 3 trial (JAKARTA) in patients with primary or secondary myelofibrosis, as well as a single-arm, open-label phase 2 trial (JAKARTA2) in patients with primary or secondary myelofibrosis previously exposed to ruxolitinib, currently the only FDA-approved treatment for the disease.
The JAKARTA trial is evaluated the efficacy of daily oral doses (400 mg or 500 mg) of fedratinib compared with placebo in patients with intermediate-2 or high-risk primary myelofibrosis, post–polycythemia vera myelofibrosis, or post–essential thrombocythemia myelofibrosis with splenomegaly. The study enrolled 289 patients in 24 countries.
Celgene also plans to study fedratinib as a potential combination treatment with luspatercept, an experimental drug currently in phase 3 clinical trials for the treatment of anemia in betathalassemia and myelodysplastic syndromes.
“The number [of patients with myelofibrosis] who are ineligible for or failed existing therapy continues to increase, representing a well-defined unmet medical need. We believe fedratinib can play an important role in the treatment of myelofibrosis and we look forward to working with the FDA as the review process advances,” said Backstrom.
The FDA has set an action date of September 3, 2019.