Fedratinib, a pyrimidine-based JAK2 and FLT3 inhibitor, has been granted approval for marketing authorization from the European Commission (EC) for the treatment of disease-related splenomegaly or symptoms in adult patients with primary myelofibrosis (MF), or secondary MF, i.e., post-polycythemia vera MF and post-essential thrombocytosis MF who are treatment naïve or have received prior ruxolitinib therapy . Fedratinib provides a once-daily, oral therapeutic option for patients.
This approval is followed by a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) given in December 2020 based on favorable results of the phase III JAKARTA and JAKARTA-2 trials, where fedratinib showed clinically meaningful reductions in spleen volume and symptoms in patients who failed ruxolitinib. These trials enrolled patients from 14 countries in the European Union.
Fedratinib is also approved by the U.S. Food and Drug Administration (FDA) and Health Canada for the treatment of adults with intermediate-2 or high-risk primary or secondary MF. Before its approval by the FDA in August 2019, fedratinib was placed on a 4-year hold due to suspected serious neurological toxicities. Read more about the approval timeline here.