FDA Grants Fast Track Designation to Devimistat for Treatment of AML

The FDA has granted fast track designation to devimistat (CPI-613) for the treatment of acute myeloid leukemia (AML), according to Rafael Pharmaceuticals, the developer of the agent.

Devimistat targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. The agent is designed to target the mitochondrial tricarboxylic acid (TCA) cycle selectively in cancer cells.

The company indicated it specifically designated devimistat as an orphan drug for the treatment of pancreatic cancer, AML, myelodysplastic syndrome, peripheral T-cell lymphoma, Burkitt’s lymphoma, and soft tissue sarcoma.

“Receiving fast track designation, especially during a pandemic that has created significant challenges for many trials across the globe, is a testament to the dedicated work of the Rafael team,” Sanjeev Luther, president and CEO of Rafael, said in a press release. “We would not be here without the support of the FDA, our doctors, our patients, and all who are invested in the hope of finding a successful treatment for this hard-to-treat cancer.”

Importantly, the FDA has already given Rafael approval to initiate pivotal phase 3 clinical trials in both pancreatic cancer (AVENGER 500®) and AML (ARMADA 2000). The EMA has also granted orphan drug designation to devimistat for pancreatic cancer and AML.

This announcement also comes on the heels of Rafael receiving fast track designation for devimistat for the treatment of metastatic pancreatic cancer in November. In addition, the company received orphan drug designation for the treatment of soft tissue sarcoma for devimistat and has also initiated a phase 2 clinical trial of devimistat in combination with hydroxychloroquine in patients with clear cell sarcoma of soft tissue.

“This designation underscores the pressing need to find new ways to combat this aggressive disease,” Jorge Cortes, MD, director of the Georgia Cancer Center at Augusta University and principal investigator on the phase 3 clinical trial of devimistat, said in the release. “It brings hope not only to clinicians, but to patients who hear that they have been diagnosed.”

Reference:

Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for the Treatment of Acute Myeloid Leukemia (AML). Cranbury, NJ. Published December 15, 2020. Accessed December 15, 2020. http://www.globenewswire.com/news-release/2020/12/15/2145445/0/en/Rafael-Pharmaceuticals-Receives-FDA-Fast-Track-Designation-for-CPI-613-devimistat-for-the-Treatment-of-Acute-Myeloid-Leukemia-AML.html