Benzinga | Oct 29, 2020 09:31AM EDT

FDA Grants Rafael Pharmaceuticals Orphan Drug Designation For CPI-613 For Treatment Of Soft Tissue Sarcoma

Company to launch clinical trial in clear cell sarcoma, a subtype of soft tissue sarcoma, in the coming months

CRANBURY, N.J., Oct. 29, 2020 (GLOBE NEWSWIRE) -- Rafael Pharmaceuticals, Inc. ("Rafael" or the "Company"), a leader in the growing field of cancer metabolism-based therapeutics announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for CPI-613(r) (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."

This announcement comes on the heels of the Company's most recent news around crossing the enrollment of 100 patients in its Phase 3 trial for relapsed or refractory acute myeloid leukemia (AML).