United States Orphan Drug Designation for Treatment of Soft Tissue Sarcomas Granted to SPEAR T-cells Targeting MAGE-A4
Adaptimmune Therapeutics plc, a leader in T-cell therapy to treat cancer, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to SPEAR T-cells targeting MAGE-A4 (Adaptimmune’s ADP-A2M4 program) for the treatment of soft tissue sarcomas. Orphan Designation by FDA was created to encourage the development of drugs for rare diseases, such as sarcomas.
“We recently started SPEARHEAD-1 treating patients with synovial sarcoma and myxoid/round cell liposarcoma (MRCLS), with the aim to launch ADP-A2M4 in 2022,” said Elliot Norry, Adaptimmune’s interim Chief Medical Officer. “This orphan drug status is another key milestone in achieving our goal of providing this much-needed therapy to people living with these rare and deadly cancers, for which there are few other treatment options.”
About soft tissue sarcomas
Soft tissue sarcomas can develop from soft tissues like fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. There are approximately 50 types of soft tissue sarcomas. Adaptimmune is currently investigating its ADP-A2M4 SPEAR T-cells in two types of sarcoma: synovial sarcoma and MRCLS.
Synovial sarcoma is a cancer of the connective tissue around joints. The most common locations are the hip, knee, ankle, and shoulder. Synovial sarcoma accounts for approximately 6% to 10% of all soft tissue sarcomas. Approximately one third of synovial sarcomas occur in childhood and the peak incidence is in the third decade of life.
Liposarcomas are another type of sarcoma, which are malignant tumors of fat tissue. Myxoid/round cell liposarcoma (MRCLS) is a malignant fat tissue sarcoma that is found predominantly in the limbs. One third of MRCLS cases will become metastatic with tumors spreading to bone and soft tissue locations. MRCLS commonly presents at an age ranging from 35-55 years and has a poor prognosis because it recurs locally and tends to metastasize quickly and widely.