FDA grants priority review to pacritinib for myelofibrosis with severe thrombocytopenia
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The FDA granted priority review to pacritinib for treatment of patients with myelofibrosis with severe thrombocytopenia, according to the agent’s manufacturer.
Approximately one-third of patients with myelofibrosis develop severe thrombocytopenia, defined as blood platelet counts less than 50 x 109/L.
Treatment options for these patients are limited, and OS is approximately 15 months.
Thrombocytopenia is associated with underlying disease; however, it also can result from treatment with ruxolitinib (Jakafi, Incyte), a Janus kinase inhibitor approved for myelofibrosis. This can lead to treatment discontinuation. Survival for patients who discontinue ruxolitinib averages 7 to 14 months.
Pacritinib (CTI BioPharma) is an investigational oral kinase inhibitor with specificity for JAK2, IRAK1 and CSF1R.
The FDA accepted a new drug application for the agent based on results of the phase 3 PERSIST-2 and PERSIST-1 trials, as well as the phase 2 PAC203 trial.
Patients with myelofibrosis and severe thrombocytopenia enrolled in these studies received 200 mg pacritinib twice daily.
In the PERSIST-2 study, a higher percentage of patients who received pacritinib than best available therapy achieved at least 35% reduction in spleen volume (29% vs. 3%) and at least a 50% reduction in total symptom scores (23% vs. 13%).
Adverse events among pacritinib-treated patients generally were low grade, manageable with supportive care and rarely led to treatment discontinuation, according to a CTI BioPharma-issued press release. Researchers also reported stabilized platelet counts and hemoglobin levels among pacritinib-treated patients.
The FDA is expected to make a decision on the application by Nov. 30.
“We are pleased that the FDA's acceptance of our NDA brings us one step closer to our goal of providing [patients with myelofibrosis] with severe thrombocytopenia a new treatment option,” Adam R. Craig, MD, PhD, president and CEO of CTI BioPharma, said in the release. “With commercial preparation underway, we believe we will be well-positioned for a potential U.S. launch later this year. We look forward to working with the FDA during its review of our application.”
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