The FDA granted priority review to fedratinib for the treatment of patients with myelofibrosis.
Fedratinib (Celgene) is a highly selective JAK2 inhibitor.
Abnormal activation of JAK2 is common among patients with myeloproliferative neoplasms, including myelofibrosis.
The new drug application for fedratinib is based on results from two trials.
The randomized phase 3 JAKARTA trial compared the agent with placebo for patients with primary or secondary myelofibrosis. The single-arm, open-label phase 2 JAKARTA2 trial included patients with primary or secondary myelofibrosis who received prior ruxolitinib (Jakafi, Incyte), the only FDA-approved therapy for myelofibrosis.
The agency previously granted orphan drug designation to fedratinib for the treatment of secondary and primary myelofibrosis.
The FDA is expected to make a decision on approval by Sept. 3.
“The acceptance of the new drug application and granting of priority review for fedratinib represent the first potential new treatment option after many years for patients affected by myelofibrosis,” Jay Backstrom, MD, chief medical officer for Celgene, said in a company-issued press release. “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.”
The FDA placed a clinical hold on the fedratinib development program in 2013 after potential cases of Wernicke’s encephalopathy were reported among approximately 1% of clinical trial participants. The FDA removed the clinical hold in 2017.