FDA approves gene therapy for synovial sarcoma

The United States Food and Drug Administration approved Tecelra, afamitresgene autoleucel, as a new gene therapy for unresectable or metastatic synovial sarcoma. More specifically, the treatment is intended for patients who have received prior chemotherapy, have tumors that express the MAGE-A4 antigen, and are HLA antigen(s) A*02:01P, –A*02:02P, –A*02:03P, or –A*02:06P positive.

“Potentially life-threatening cancers such as synovial sarcoma continue to have a devastating impact on individuals, especially those for whom standard treatments have limited efficacy due to tumor growth and progression,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research (CBER). “The approval of this state-of-the-art immunotherapy technology provides a critical new option for a patient population in need and demonstrates the FDA’s dedication to the advancement of beneficial cancer treatments.”

Synovial sarcoma is a rare cancer that generally impacts 1,000 people in the US annually. According to the FDA, this type of cancer causes malignant cells to develop and tumor formation in the soft tissues, often affecting the larger joints in the body.

“Adults with metastatic synovial sarcoma, a life-threatening form of cancer, often face limited treatment options in addition to the risk of cancer spread or recurrence,” said Nicole Verdun, MD, director of the Office of Therapeutic Products in CBER. “Today’s approval represents a significant milestone in the development of an innovative, safe, and effective therapy for patients with this rare but potentially fatal disease.”

Tecelra is the first FDA-approved T cell receptor gene therapy. The treatment modifies a patient’s own T cells to express a T cell receptor that targets the MAGE-A4 antigens expressed on synovial sarcoma cancer cells. The product is then administered intravenously.

The FDA approval was based on a multi-center, open-label clinical trial that supported the drug's safety and efficacy. The FDA press release notes that the overall response rate was 43.2%. The most commonly reported side effects were nausea, vomiting, fatigue, infections, fever, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, and edema.