FDA Approves First Cell Therapy for Solid Tumor

Rare Daily Staff

The U.S. Food and Drug Administration granted accelerated approval to Adaptimmune’s Tecelra for a rare cancer, making it the first approved engineered cell therapy for a solid tumor.

The FDA approved Tecelra for advanced MAGE-A4+synovial sarcoma in adults with certain HLA types who have received prior chemotherapy. It is the first new treatment option for people with synovial sarcoma in more than a decade

There are more than 50 different types of soft tissue sarcomas which are categorized by tumors that appear in fat, muscle, nerves, fibrous tissues, blood vessels, or deep skin tissues. Synovial sarcoma accounts for approximately 5 percent to 10 percent of all soft tissue sarcomas (there are approximately 13,400 new soft tissue cases in the United States each year). One-third of patients with synovial sarcoma will be diagnosed under the age of 30. The five-year survival rate for people with metastatic disease is approximately 20 percent, and most people undergoing standard-of-care treatment for advanced disease experience recurrence and go through multiple lines of therapy, often exhausting all options.

Tecelra is a melanoma-associated antigen A4 (MAGE-A4)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adults with unresectable or metastatic synovial sarcoma who have received prior chemotherapy, are HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and whose tumor expresses the MAGE-A4 antigen as determined by FDA-approved or cleared companion diagnostic devices.

Tecelra can cause serious side effects, including cytokine release syndrome, immune effector cell–associated neurotoxicity syndrome, prolonged severe cytopenia, infections, secondary malignancies, and hypersensitivity reactions. The most common adverse reactions (incidence ≥20%) include cytokine release syndrome, nausea, vomiting, fatigue, infections, pyrexia, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, edema, low white blood cell counts, low red blood cell counts and low platelets.

The agency granted accelerated approval for the indication based on overall response rate and duration of response. Continued approval for the indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

“For decades, therapeutic options for people diagnosed with synovial sarcoma have been limited. It is long past time that synovial sarcoma patients have expanded treatment options,” said Brandi Felser, CEO of the Sarcoma Foundation of America. “Since one-third of patients are diagnosed under age 30, improved outcomes can have a tremendous impact.”

Photo: Brandi Felser, CEO of the Sarcoma Foundation of America