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The FDA granted (afami-cel, Tecelra) for treating advanced synovial sarcoma, the first engineered cell therapy indicated for a solid tumor.

An autologous T-cell immunotherapy, afami-cel is specifically indicated for adults with unresectable or metastatic disease who have received prior chemotherapy. For eligibility, patients need to be HLA-A*02:01P, -A*02:02P, -A*02:03P, or -A*02:06P positive and have a tumor that expresses the MAGE-A4 antigen, as determined by a companion diagnostic.

With the approval, the T-cell receptor gene therapy becomes the first new therapeutic option in over a decade for synovial sarcoma, a rare soft-tissue cancer that most commonly develops in the extremities.

"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," Peter Marks, MD, PhD, director of FDA's Center for Biologics Evaluation and Research, said in a statement.

Approval was based on results from cohort 1 of the SPEARHEAD-1 trial, which included 44 patients with advanced synovial sarcoma. One-time treatment with afami-cel resulted in an overall response rate of 43.2% and a complete response rate of 4.5%. Median duration of response was 6 months, and 39% of responders had a response lasting a year or longer.

"Tecelra, which uses each patient's own immune cells to recognize and attack their cancer cells in a one-time infusion treatment, is significantly different than the current standards of care for advanced synovial sarcoma," said investigator Sandra D'Angelo, MD, of Memorial Sloan Kettering Cancer Center in New York City, in a from drugmaker Adaptimmune.

Synovial sarcoma is diagnosed in about 1,000 people in the U.S. annually, most often adult males in their 30s or younger. Most patients receiving standard treatments for advanced disease have recurrence and go through many lines of therapy, often exhausting all options. The 5-year survival rate for those with metastatic disease is around 20%.

"This approval represents a much-needed new option for people diagnosed with this sarcoma and an important milestone for the use of cell therapies in solid tumor cancers," said D'Angelo.

Similar to CAR T-cell therapies, carries a boxed warning for cytokine release syndrome (CRS). In trials, CRS occurred in 75% of patients (2% grade ≥3) with a median onset of 2 days and median resolution of 3 days; common symptoms of CRS included fever, tachycardia, hypotension, nausea/vomiting, and headache. The side effect was managed with tocilizumab (Actemra) in 55% of patients who experienced CRS.

Beyond CRS, common adverse events (incidence ≥20%) with afami-cel included nausea, vomiting, fatigue, infections, pyrexia, constipation, dyspnea, abdominal pain, non-cardiac chest pain, decreased appetite, tachycardia, back pain, hypotension, diarrhea, and edema, along with low counts of white blood cells, red blood cells, and platelets.

Immune effector cell-associated neurotoxicity syndrome was observed in one patient following administration of afami-cel (onset in 2 days, resolution after 1) and is included among the warnings and precautions in the prescribing information. Other warnings include risks for severe cytopenia, secondary malignancies, infections, and effects on driving or operating machinery.

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