FDA Grants Rafael Pharmaceuticals Orphan Drug Designation for CPI-613

Rafael Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for CPI-613® (devimistat) for the treatment of soft tissue sarcoma.

Soft tissue sarcoma is a type of cancer that starts in the soft tissues of the body, such as fat, muscle, nerves, fibrous tissues, blood vessels or deep skin tissues. There are more than 50 different types of soft tissue sarcomas, and clear cell is ranked among the rarest. Rafael’s clinical trial will focus on the treatment of relapsed or refractory clear cell sarcoma.

“There is a significant unmet need in treatment for soft tissue sarcoma,” said Sanjeev Luther, President and CEO of Rafael Pharmaceuticals. “When a disease is rare, it often does not receive the attention and focus necessary to develop effective treatments for it. We want to be a voice for those diagnosed with hard-to-treat cancers so that they know that they are not forgotten. We are focusing our attention on developing treatments for these cancers. In fact, Rafael is one of the only companies working on a treatment for clear cell sarcoma.”

CPI-613® (devimistat) is a first-in-class clinical lead compound of Rafael, which targets enzymes that are involved in cancer cell energy metabolism and are located in the mitochondria of cancer cells. Devimistat is designed to target the mitochondrial tricarboxylic acid (TCA) cycle, a process essential to tumor cell multiplication and survival, selectively in cancer cells. Devimistat substantially increases the sensitivity of cancer cells to a diverse range of chemotherapeutic agents. This synergy allows for potential combinations of devimistat with lower doses of these generally toxic drugs to be more effective with lower patient’s side effects. Combination with devimistat represents a diverse range of opportunities to substantially improve patient’s benefit in many different cancers. The U.S. Food and Drug Administration (FDA) has given Rafael approval to initiate pivotal Phase 3 clinical trials in pancreatic cancer (AVENGER 500®) and acute myeloid leukemia (ARMADA 2000), and has designated devimistat as an orphan drug for the treatment of pancreatic cancer, acute myeloid leukemia, myelodysplastic syndrome, peripheral T-cell lymphoma and Burkitt’s lymphoma. The EMA has granted orphan drug designation to devimistat for pancreatic cancer and acute myeloid leukemia.