The FDA has accepted the New Drug Application and granted a Priority Review for fedratinib for the treatment of patients with myelofibrosis, according to the agent’s manufacturer.

Fedratinib, under development from Celgene, is a highly selective JAK2 inhibitor.

“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.” Jay Backstrom, MD, Chief Medical Officer for Celgene, said in a press release. “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.”

Woman holding a pill. Source: Getty

The FDA based the priority review designation on results from the randomized, phase 3 JAKARTA trial and the open-label, single-arm phase 2 JAKARTA2 trial. The studies assessed patients with primary or secondary myelofibrosis. The JAKARTA2 trial assessed patients with previous exposure to ruxolitinib, which is the only FDA-approved treatment for myelofibrosis.

JAKARTA included 289 patients with intermediate-2 or high-risk primary myelofibrosis, post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis with splenomegaly from 24 countries.

JAKARTA2 included 97 patients from 40 sites in 10 countries.

Spleen response rate, defined as the proportion of patients who had a reduction in spleen volume of at least 35% after six one-month treatment cycles, served as the primary endpoint in both trials. Secondary endpoints included symptom response rates.