The FDA recently granted accelerated approval to afamitresgene autoleucel (afami-cel, Tecelra) for the treatment of advanced synovial sarcoma. This marks a significant milestone as afami-cel is the first engineered cell therapy indicated for a solid tumor. Synovial sarcoma is a rare soft-tissue cancer that predominantly affects individuals with unresectable or metastatic disease who have received previous chemotherapy treatments. The eligibility criteria for receiving afami-cel includes being HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and having a tumor that expresses the MAGE-A4 antigen. These requirements are determined by a companion diagnostic to ensure the therapy is suitable for the patient.
The approval of afami-cel was based on results from cohort 1 of the SPEARHEAD-1 trial, which included 44 patients with advanced synovial sarcoma. The data showed that a one-time treatment with afami-cel resulted in an overall response rate of 43.2% and a complete response rate of 4.5%. The median duration of response was 6 months, with 39% of responders having a response lasting a year or longer. This is a significant development as it provides a new therapeutic option after more than a decade for individuals diagnosed with synovial sarcoma.
Similar to CAR T-cell therapies, afami-cel comes with potential risks and side effects that patients need to be aware of. One of the notable side effects is cytokine release syndrome (CRS), which occurred in 75% of patients during trials. The common symptoms of CRS include fever, tachycardia, hypotension, nausea/vomiting, and headache. To manage CRS, tocilizumab (Actemra) was used in 55% of patients who experienced the syndrome. Other common adverse events associated with afami-cel include nausea, vomiting, fatigue, infections, pyrexia, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, and edema. Additionally, low counts of white blood cells, red blood cells, and platelets were observed in some patients.
The FDA’s approval of afamitresgene autoleucel (afami-cel) represents a significant advancement in the treatment of advanced synovial sarcoma. This new therapy option offers hope to individuals diagnosed with this rare soft-tissue cancer, especially those who have exhausted standard treatment options. While the therapy has shown promising results in clinical trials, patients need to be aware of the potential risks and side effects associated with afami-cel, including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome. It is important for healthcare providers and patients to weigh the benefits and risks of afami-cel before initiating treatment for synovial sarcoma.
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