Adaptimmune Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, FDA granted Orphan Drug Designation (ODD) to ADP-A2M4 for the treatment of soft tissue sarcomas.
“RMAT designation is another important step in bringing our ADP-A2M4 therapy to market in 2022 for patients with synovial sarcoma,” said Elliot Norry, Adaptimmune’s acting Chief Medical Officer. “We have compelling data with ADP-A2M4 for the treatment of synovial sarcoma and are eager to make this therapy available to patients who have few other treatment options. We are screening and enrolling patients with sarcoma in our Phase 2 SPEARHEAD-1 trial.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products. A product is eligible for RMAT designation if it is a regenerative medicine therapy, such as a T-cell therapy, and is intended to treat, modify, reverse or cure a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs for such disease or condition.
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RMAT designation includes the incentives of Breakthrough Therapy designation including additional FDA interaction and guidance, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA) and other opportunities to expedite development and review.
Data from patients with synovial sarcoma treated in the expansion phase of Adaptimmune’s Phase 1 trial with ADP-A2M4 were recently presented at CTOS. There was an overall response rate of 50%, and a disease control rate of 93% with 13 out of 14 patients showing clinical benefit with best overall responses of partial responses (confirmed or unconfirmed; n=7) or stable disease (n=6).