The U.S. Food and Drug Administration (FDA) has accepted for priority review Adaptimmune Therapeutics plc's Biologics License Application (BLA) for afami-cel, an investigational engineered T-cell therapy aimed at treating advanced synovial sarcoma. This acceptance is a pivotal moment in the journey towards providing a novel treatment option for patients suffering from this type of cancer. The Prescription Drug User Fee Act (PDUFA) target action date is set for August 4, 2024.
Afami-cel represents a groundbreaking approach as the first engineered T-cell therapy for solid tumors and the first effective treatment option for synovial sarcoma in over a decade. The therapy targets the MAGE A4 cancer target and is designed as a single-dose treatment. The BLA submission is supported by positive data from Cohort 1 of the pivotal trial SPEARHEAD-1, which demonstrated an impressive response rate of approximately 39% among heavily pre-treated patients with advanced synovial sarcoma and a median survival of about 17 months.
Synovial sarcoma, accounting for approximately 5% to 10% of all soft tissue sarcomas, presents significant treatment challenges, with a five-year survival rate for metastatic disease being just 20%. The acceptance of afami-cel's BLA by the FDA is a testament to the potential of this therapy to change the treatment landscape for patients with synovial sarcoma, offering hope for improved outcomes and survival rates.
Adaptimmune is at the forefront of redefining cancer treatment with its unique engineered T-cell receptor (TCR) platform, which enables the engineering of T-cells to target and destroy cancers across multiple solid tumor types. The company's commitment to advancing TCR therapeutic candidates through the regulatory and commercialization processes underscores its dedication to improving the lives of patients and their families.
