Celgene Corporation today announced the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for fedratinib and granted a Priority Review. Fedratinib is a highly selective JAK2 inhibitor intended for the treatment of patients with myelofibrosis, a serious bone marrow disorder that disrupts the body’s normal production of blood cells. Under the Prescription Drug User Fee Act, the FDA has set its action date as Sept. 3, 2019.
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. Patients with myelofibrosis, including the number 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 Jay Backstrom, M.D.,
Chief Medical Officer for Celgene.
The NDA for fedratinib is 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, the only FDA-approved treatment for the disease. Results of these two trials have been previously published in peer-reviewed journals. The FDA has also provided fedratinib Orphan Drug designation for the treatment of secondary and primary myelofibrosis.
Celgene also plans to evaluate fedratinib in combination with luspatercept.
Fedratinib is an investigational compound that is not approved for any use in any country.