Pharma Focus Asia

C4 Therapeutics Announces FDA Orphan Drug Designation for CFT7455 for the Treatment of Multiple Myeloma

Thursday, August 12, 2021

C4 Therapeutics, Inc. (C4T), a clinical-stage biopharmaceutical company pioneering a new class of small-molecule medicines that selectively destroy disease-causing proteins through degradation, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT7455 for the treatment of multiple myeloma.  

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.

“We are pleased to receive FDA’s orphan drug designation for CFT7455 in multiple myeloma and believe this designation highlights the potential of CFT7455 to improve clinical outcomes for patients with multiple myeloma who face an incurable disease,” said Adam Crystal, M.D., Ph.D., chief medical officer of C4 Therapeutics. “With far too many patients relapsing on numerous lines of therapy and succumbing to multiple myeloma, we are focused on advancing our Phase 1/2 trial to bring this new treatment option to patients.”

CFT7455 is an orally bioavailable MonoDAC™ degrader targeting IKZF1/3 for the treatment of multiple myeloma and non-Hodgkin’s lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma. In June 2021, C4T initiated the Phase 1/2 clinical trial to primarily investigate safety, tolerability, and anti-tumor activity, with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT7455. Across the Phase 1/2 trial, C4T plans to enroll approximately 160 patients.

magazine-slider-imageMFA + MMA 2024CPHI Chine || PMEC China 2024Asia Healthcare Week 2024Advance DoE WorkshopNitrosamine Advance Workshop 2024CPHI Korea 2024CHEMICAL INDONESIA 2024INALAB 2024 Thermo Scientific - DynaDrive and DynaSpinDigital Health Asia 2024Rehab Expo 2024ISPE Singapore Affiliate Conference & Exhibition 20242024 PDA Pharmaceutical Manufacturing & Quality Conference2024 PDA Cell and Gene Pharmaceutical Products Conference 2024 PDA Aseptic Manufacturing Excellence Conference2024 PDA Aseptic Processing of Biopharmaceuticals ConferencePharma Quality Excellence Awards 20244th Annual Pharma GMP Quality Management 20243rd World ADC Asia 2024LogiPharma Asia 2024