The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) for fedratinib (Celgene), an investigational agent being developed for the treatment of myelofibrosis (MF).

Fedratinib is an oral kinase inhibitor with activity against wild type and mutationally activated Janus Associated Kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3). The NDA is supported by results from the JAKARTA (phase 3) and JAKARTA2 (phase 2) trials.

In the phase 3 trial, 289 patients with intermediate-2 or high-risk primary MF, post-polycythemia vera MF, or post-essential thrombocythemia MF were randomized to receive fedratinib or placebo for at least 6 consecutive 4-week cycles. Results showed that fedratinib significantly reduced splenomegaly and symptom burden in patients with MF, but was associated with toxic effects (encephalopathy of unknown mechanism) in some patients.

Cases of Wernicke’s encephalopathy put the clinical program for fedratinib on hold in November 2013, however the FDA removed the hold in August 2017 following a review of the data. A Prescription Drug User Fee Act target data for the NDA has been set for September 3, 2019.

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