A Philly biotech has earned FDA approval for the first-of-its-kind cancer therapy

A biotechnology company located at the Navy Yard received approval Thursday from the U.S. Food and Drug Administration for a novel type of cancer therapy.

The therapy, manufactured by Adaptimmune, targets synovial sarcoma, a rare form of cancer that appears in soft tissue, such as muscles and ligaments. Roughly 1,000 U.S. residents have synovial sarcoma. Most patients are men, one-third of whom are diagnosed before they turn 30.

Because relatively few treatment options for synovial sarcoma exist, only one-third of patients are alive five years after diagnosis. The new therapy, Tecelra, is the first approved for synovial sarcoma in more than 10 years.

“Today, there is a renewed sense of hope for this patient community,” Brandi Felser, chief executive officer of the Sarcoma Foundation of America, said in a statement.

The FDA signed off on Tecelra as part of an “accelerated approval pathway,” in which patients can receive the treatment while the company continues to test its effectiveness and safety, a process that can take years.

The government agency based its approval on early data published in March in The Lancet. In an Adaptimmune-funded trial of 44 people with synovial sarcoma, roughly 40% responded to Tecelra, which is administered as one intravenous infusion. Two people had a “complete response,” meaning there was no sign of the disease.

“The approval of this state-of-the-art immunotherapy technology provides a critical new option for a patient population in need and demonstrates the FDA’s dedication to the advancement of beneficial cancer treatments,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.

Tecelra is the first type of therapy of its kind, and the first to target solid tumors — which represent 90% of cancers in adults — using modified immune cells known as T cells. As part of the therapy, scientists extract the patient’s own T cells, then tweak the cells to target the synovial sarcoma cancer cells, and inject them back into the body.

Tecelra is one of many ways researchers are exploring how to train the body’s immune system to fight cancer. Along with Tecelra, the FDA has given a green light to several therapies that also modify a patient’s own T cells to target markers on the surface of cancer cells.

For instance, chimeric antigen receptor T cell therapy, or CAR-T therapy, was pioneered at the University of Pennsylvania, and has been heralded as a cure for some forms of blood cancer. But CAR-T therapy doesn’t work well against solid tumors.

Since Tecelra can target a marker associated with a solid tumor, the latest approval suggests the new treatment may hold promise for treating cancers that CAR-T therapies cannot reach.

During the Tecelra trial, which also included eight patients with another type of cancer that originates in fat cells, the majority of patients experienced cytokine release syndrome, a potentially dangerous overreaction by the immune system to the treatment. None died as a result.

“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 Sandra D’Angelo, a researcher based at Memorial Sloan Kettering Cancer Center in New York, and the lead investigator in The Lancet trial, in a statement.

Adaptimmune, which also has offices in Massachusetts and the United Kingdom, says it is ready to accept orders for Tecelra, and plans to establish at least six to 10 treatment centers this year, and 30 within two years. The FDA has only approved it for patients who have already tried chemotherapy, and whose tumors express the specific marker the therapy is designed to target. The price is $700,000 for one infusion.

To maintain its FDA approval, the company must continue to study the treatment and confirm its benefits.