On March 9, 2022 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT8634 for the treatment of soft tissue sarcoma (Press release, C4 Therapeutics, MAR 9, 2022, View Source [SID1234609962]).
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The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.
"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. 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," said Adam Crystal, M.D., Ph.D., chief medical officer of C4 Therapeutics.
CFT8634 is a BiDAC degrader targeting BRD9 for the treatment of cancers that are dependent on BRD9, including synovial sarcoma and SMARCB1 deleted cancers. BRD9 has been considered an "undruggable" target because inhibitors of the bromodomain of BRD9 are not effective in treating these cancers. However, C4T’s TORPEDO platform was leveraged to discover CFT8634, an orally bioavailable, selective degrader of BRD9. Unlike BRD9 inhibition, BRD9 degradation is efficacious in preclinical models of synovial sarcoma. In December 2021, the FDA cleared C4T’s investigational new drug (IND) application for CFT8634 to proceed with the proposed Phase 1/2 trial in patients with synovial sarcoma and SMARCB1-null solid tumors. Site activation efforts have commenced and the trial remains on track to begin dosing patients in the first half of 2022.