The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to pracinostat in combination with azacitidine for the treatment of patients with newly-diagnosed acute myeloid leukemia (AML) who are older than 75 years or unfit for intensive chemotherapy, according to a news release.1

The designation was granted because of results from a phase 2 study of pracinostat plus azacitidine in elderly patients who were not candidates for induction chemotherapy. Patients had a median overall survival of 19.1 months and a complete response rate of 42%.

The results were found to have compared favorably to findings from a phase 3 study of azacitidine, which showed a median overall survival of 10.4 months with azacitidine alone and a complete response rate of 19.5% in a similar patient population. Combination pracinostat and azacitidine was found to be generally well-tolerated with no unexpected toxicities.

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MEI Pharma reached an agreement with the FDA on a proposed phase 3 study design.

“This designation speaks to both the serious unmet need for AML patients unfit to receive intensive chemotherapy and the promise of pracinostat to address this need,” said Daniel Gold, PhD, president and chief executive officer of MEI Pharma.


  1. MEI Pharma’s pracinostat receives breakthrough therapy designation from FDA for treatment in combination with azacitidine of patients with newly diagnosed acute myeloid leukemia unfit for intensive chemotherapy. PRNewswire. Updated August 1, 2016. Accessed August 1, 2016.