Celgene has secured the US Food and Drug Administration (FDA) priority review status for the new drug application (NDA) of fedratinib to treat patients with myelofibrosis.
Myelofibrosis is a serious bone marrow disorder that affects the normal production of blood cells.
Fedratinib is an oral kinase inhibitor targeting wild type and mutationally activated janus associated kinase 2 (JAK2) and FMS-like tyrosine kinase 3 (FLT3).
Abnormal JAK2 activation is linked to myeloproliferative neoplasms (MPNs), including myelofibrosis.
The drug led to decrease in phosphorylation of signal transducer and activator of transcription (STAT3/5) proteins, inhibited cell proliferation and triggered apoptotic cell death in cell models expressing mutationally active JAK2 or FLT3.
Celgene chief medical officer Jay Backstrom said: “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, represent a well-defined unmet medical need.”
The NDA submitted to the FDA is supported by data from the Phase III JAKARTA and Phase II JAKARTA2 trials conducted in primary or secondary myelofibrosis patients.
The multi-centre, randomised, double-blind, placebo-controlled JAKARTA trial assessed 400mg and 500mg daily oral doses of fedratinib in 289 participants at 94 sites across 24 countries.
During the multi-centre, open label, single-arm JAKARTA2 trial, 400mg daily fedratinib dose was evaluated in 97 patients across 40 centres in ten countries. This trial enrolled patients previously treated with ruxolitinib.
Primary endpoint of the trials was proportion of patients with at least 35% decrease in spleen volume following six one-month treatment cycles. Secondary endpoints included symptom response rate, the number of patients with a 50% or greater reduction in Total Symptom Score after six one-month treatment cycles.
Celgene is also planning to further study fedratinib in combination with luspatercept.