FDA grants priority review to filing for Celgene's JAK2 inhibitor fedratinib in myelofibrosis

Celgene on Tuesday announced that the FDA granted priority review to a marketing application seeking approval of the JAK2 inhibitor fedratinib for the treatment of patients with myelofibrosis, setting a target action date of September 3. The company gained rights to the therapy, which is also being developed to treat polycythaemia vera, as part of its acquisition of Impact Biomedicines last year under a deal potentially worth up to $7 billion. 

Celgene noted that its submission for fedratinib in the myelofibrosis indication is supported by data from the Phase III JAKARTA study of patients with primary or secondary myelofibrosis. Results showed that fedratinib met the primary and secondary endpoints by reducing spleen size in 47 percent of patients by at least 35 percent at 24 weeks, while also improving symptom scores in 36 percent of patients by at least 50 percent, versus placebo. The filing is also backed by results from the mid-stage JAKARTA-2 study of patients with primary or secondary myelofibrosis previously exposed to Incyte's Jakafi (ruxolitinib), which the FDA approved in 2011 to treat the disease.  

Sanofi halted development of fedratinib in 2013 after the compound was linked to "cases consistent with Wernicke's encephalopathy" in patients participating in fedratinib trials, with the FDA placing a clinical hold on studies of the therapy. Impact Biomedicines later acquired full rights for the global development and commercialisation of fedratinib from Sanofi, and the FDA lifted the clinical hold on trials of the drug in 2017.

Celgene chief medical officer Jay Backstrom said "patients with myelofibrosis, including the number who are ineligible for or failed existing therapy continues to increase, representing a well-defined unmet medical need." He added "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 drug was previously granted orphan drug status by the agency for the treatment of patients with primary or secondary myelofibrosis. Celgene also expressed plans to study fedratinib in combination with the experimental drug luspatercept, which the company is co-developing with Acceleron Pharma to treat beta-thalassaemia-associated anaemia and myelodysplastic syndromes.

Earlier this year, Celgene entered into a $74-billion agreement to be acquired by Bristol-Myers Squibb (for related analysis, see ViewPoints: The hunted goes hunting – for liberty and a late-stage pipeline). Wellington Management, Bristol-Myers Squibb's largest shareholder, recently came out against the deal, while activist investor Starboard Value has nominated five directors to Bristol-Myers Squibb's board ahead of an April 12 shareholder vote on the proposed Celgene merger. For additional analysis, read ViewPoints: Bristol-Myers Squibb and Celgene's best laid plans start to unravel.

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