FDA Approves Unique Cell Therapy for Rare Soft Tissue Cancer

The Food and Drug Administration (FDA) has granted approval to a new cellular therapy developed by Adaptimmune for treating synovial sarcoma, a rare form of soft tissue cancer. This therapy, now officially named Tecelra, was previously referred to as afami-cel. The FDA's accelerated approval permits its use in certain patients with metastatic synovial sarcoma who have undergone chemotherapy and have specific immune markers, including the presence of the MAGE-A4 protein that Tecelra targets.

The FDA's decision was primarily based on Tecelra's ability to induce tumor responses in approximately 43% of patients during clinical trials, with these responses lasting a median of six months. To maintain this approval, Adaptimmune must confirm these benefits through an ongoing study, with results expected to be submitted next year.

Tecelra is priced at $727,000 per treatment, making it the most expensive single-dose cellular therapy for cancer in the United States. The cost reflects the clinical value it offers to patients with this rare and aggressive tumor type, according to Adaptimmune’s Chief Commercial Officer, Cintia Piccina.

Synovial sarcoma is among the rarest types of tumors, with the American Cancer Society estimating that 13,590 people will be diagnosed with soft tissue cancers in the U.S. this year. Synovial sarcomas account for roughly 5% to 10% of these cases, primarily affecting men in their 30s or younger. Treatment options are limited, and the median five-year survival rate for metastatic cases is around 20%.

Peter Marks, head of the FDA's office responsible for evaluating gene and cell therapies, emphasized the significance of this new immunotherapy, underlining the FDA's commitment to advancing effective cancer treatments. Despite the promising results, Adaptimmune has yet to prove that its treatment can extend patients' lives. Preliminary data suggests a two-year survival probability of about 70% for patients responding to the therapy.

The approval of Tecelra represents a significant advancement in the field of cell therapy, particularly as it is the first time an engineered cell therapy has been approved for a solid tumor. Unlike previous FDA-approved CAR-T therapies, which target proteins on the surface of cells, Tecelra is a TCR cell therapy that can recognize intracellular targets, potentially making it more effective against solid tumors.

Tecelra is also the first new treatment for synovial sarcoma in over a decade. Adaptimmune's CEO, Adrian Rawcliffe, hailed the FDA's approval as a watershed moment for the company. However, the journey to this point has been challenging, marked by lost partnerships, layoffs, and pipeline restructuring. Despite the approval, investor skepticism remains, reflected in the company's low share price.

Analysts at Mizuho Securities have projected peak annual sales for Tecelra at $174 million, anticipating conservative uptake. Like other cell therapies, Tecelra will only be available at authorized treatment centers equipped to administer it. Adaptimmune expects between six to ten centers to be operational this year, expanding to 30 within two years. The company anticipates the first commercial sales in the fourth quarter, with modest growth expected before increasing in 2025.

Adaptimmune views Tecelra as the beginning of a broader venture into soft tissue cancer treatments. The company plans to submit another therapy, lete-cel, for regulatory approval next year for two types of sarcomas. Combined, these therapies could potentially generate $400 million in annual peak sales in the U.S., with further growth expected if they gain approval in other regions and for additional sarcomas and treatment stages. CEO Rawcliffe expressed optimism about expanding the use of these therapies over time.

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