Regenerative Medicine Advanced Therapy Designation Granted to ADP-A2M4 for Synovial Sarcoma

Web Exclusives - FDA Updates, In the News, Synovial Sarcoma

On December 3, 2019, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to the biopharmaceutical company Adaptimmune Therapeutics for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, the FDA granted Orphan Drug designation to the agent for the treatment of soft tissue sarcomas.

RMAT designation was established under the 21st Century Cures Act to expedite the drug development and review processes for promising new products. To be eligible for this designation, a product must be a regenerative medicine therapy that is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, with the potential to address unmet medical needs of patients with the disease or condition, based on preliminary clinical evidence.

The RMAT designation for ADP-A2M4 is based on results from the expansion phase of a phase 1 clinical trial, which were presented at the 2019 Connective Tissue Oncology Society Annual Meeting in Tokyo, Japan. In patients with synovial sarcoma who were treated with ADP-A2M4, there was an overall response rate of 50%, and a disease control rate of 93%, with 13 of 14 patients demonstrating clinical benefit with best overall responses of partial responses or stable disease.

Synovial sarcoma is a rare type of cancer that accounts for 5% to 10% of soft-tissue tumors. It affects the soft tissues, such as muscle or ligaments, fat, blood or lymph vessels, nerves, and tendons that connect, support, and surround bones and organs. It tends to develop near large joints, particularly the knee, in young adults between the ages of 15 and 40 years.

In a company press release, Elliot Norry, MD, Adaptimmune’s Acting Chief Medical Officer said, “RMAT designation is another important step in bringing our ADP-A2M4 therapy to market in 2022 for patients with synovial sarcoma […] and we are eager to make this therapy available to patients who have few other treatment options.” He added, “This updated data set confirms that ADP-A2M4 continues to deliver clinical benefit, including RECIST responses, to patients with synovial sarcoma. It has also galvanized the support we have received from the sarcoma community, allowing us to rapidly open multiple sites and screen patients for the Phase 2 SPEARHEAD-1 trial.”

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Last modified: December 11, 2019