FDA Grants Orphan Drug Designation to Soft Tissue Sarcoma Treatment

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C4 Therapeutics has been granted orphan drug designation by FDA for CFT8634 for the treatment of soft tissue sarcoma.

C4 Therapeutics announced on March 9, 2022 that FDA has granted orphan drug designation (ODD) to CFT8634 for the treatment of soft tissue sarcoma. The company specializes in developing a new generation of small-molecule medicines to change how diseases are treated.

CFT8634 targets cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1 deleted cancers. It is orally bioavailable, selective degrader of BRD9, which was formerly considered an “undruggable” target because inhibitors of the bromodomain of BRD9 are not effective in treating cancers.

Orphan drug designation is granted to treatments for rare diseases that affect fewer than 200,000 people in the United States. The treatments are intended for the safe and effective treatment of patients with unmet medical needs due to these rare diseases.


“Patients living with synovial sarcoma currently have limited treatment options. After first-line treatment with chemotherapy, the benefit of which is typically of limited durability, patients with metastatic synovial sarcoma tend to do poorly,” said Adam Crystal, M.D., Ph.D., and chief medical officer of C4 Therapeutics, in a press release. “The FDA’s decision to grant orphan drug designation to CFT8634 is an important recognition of the potential of our targeted protein degrader to address this dire unmet medical need faced by patients and their families.”

Source: C4 Therapeutics