First gene therapy for metastatic synovial sarcoma earns FDA's OK!

According to the latest press release by the FDA in Aug 2024, Afamitresgene autoleucel, a novel gene therapy has been approved for people with metastatic synovial sarcoma with limited treatment options. This first-of-its-kind T cell receptor (TCR) gene therapy, designed to target the MAGE-A4 antigen present in tumour cells, provides a new lifeline for patients with this rare and aggressive cancer.

Synovial sarcoma, a malignant cancer affecting soft tissues (such as muscles and ligaments), predominantly impacts young adult males and is notoriously difficult to treat once it spreads. It often develops in the extremities but can affect different body parts and spread to other areas. Nearly 1,000 people in the U.S. are affected with this disorder yearly, mainly affecting men aged 30 years or less. In case of large, recurring or metastasized tumours, surgery, with radiotherapy or chemotherapy is used commonly.

The safety and effectiveness of this therapy have been evaluated in a clinical trial involving patients with inoperable or metastatic synovial sarcoma. These patients had previously undergone systemic treatment and whose tumours expressed the MAGE-A4 antigen. The overall response rate was 43.2%, with a median response period of 6 months among the 44 patients treated. Nausea, vomiting, fatigue, dyspnea, infections, fever, constipation, pain in the abdomen, non-cardiac chest pain, reduced appetite, tachycardia, back pain, hypotension, diarrhoea and oedema are the typical adverse events reported with Afamitresgene autoleucel.

Approved under the FDA's Accelerated Approval pathway, Afamitresgene autoleucel is lauded as a significant advancement in cancer treatment, though it comes with risks, including cytokine release syndrome and neurotoxicity. Patients are advised to be closely monitored post-treatment.

This approval highlights the FDA’s commitment to pioneering therapies that address unmet medical needs in life-threatening diseases.