FDA grants orphan drug designation to T-cell therapy for soft tissue sarcoma

The FDA granted orphan drug designation to an affinity enhanced T-cell therapy targeting NY-ESO to treat soft tissue sarcoma.

Evaluation of NY-ESO therapy (Adaptimmune) — engineered to target and destroy cancer cells by strengthening a patient’s natural T-cell response — is expected to be initiated in pivotal studies in synovial sarcoma by the end of this year. The manufacturer also plans to evaluate the therapy’s use for myxoid/round-cell liposarcoma, according to a press release.

“Soft tissue sarcomas are among the most aggressive forms of cancers, often affecting a young patient population and, for patients with metastatic and recurrent disease, therapeutic options are limited,” Rafael Amado, MD, chief medical officer of Adaptimmune, said in a press release. “We are pleased that the FDA recognizes the significance of the unmet medical need in these rare cancers, and we look forward to working with them further to expeditiously advance our T-cell therapy targeting NY-ESO through clinical development in this disease.”

The FDA Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. The designation allows manufacturers to qualify for various incentives, including tax credits for qualified clinical trials and — upon regulatory approval — 7 years of market exclusivity.

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