FDA grants anti-BCMA CAR-T orphan drug designation for advanced multiple myeloma
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The FDA granted orphan drug designation to CART-ddBCMA, a chimeric antigen receptor T-cell therapy, for the treatment of multiple myeloma, according to a press release from the manufacturer.
CART-ddBCMA (Arcellx) is an investigational autologous T-cell therapy with a novel binding domain that targets the B-cell maturation antigen (BCMA) on the surface of cancer cells. Arcellx announced in February that CART-ddBCMA had been granted FDA fast track designation for patients with relapsed or refractory multiple myeloma.
“Receiving both orphan and fast track designations further validates the potential of Arcellx technology and our novel binding domain,” Amy B. Fix, senior vice president of regulatory affairs at Arcellx, said in a company-issued press release. “We hope to provide patients with a new therapy that may significantly improve treatment response for this debilitating disease.”
The investigational therapy’s binding domain was developed using Arcellx’s proprietary platform in which antigen receptor complex T cells (ARC-T) are controlled by a tumor-targeting protein called a sparX (soluble protein antigen-receptor X-linker).
“We are pleased to be one of the first sites to test this new technology in the clinic,” Matthew Frigault, MD, assistant director of the cellular therapy service at Massachusetts General Hospital Cancer Center, member of the Cell Therapy Next Peer Perspective Board, and primary investigator of the phase 1 trial evaluating CART-ddBCMA, said in the release.
“The novel deimmunized binding domain utilized in this trial may be potentially less immunogenic than the single-chain variable fragment or camelid binders used in conventional CAR T-cell therapies,” he added.
The phase 1, open-label trial (NCT04155749) of CART-ddBCMA will be “the first in a series of clinical trials planned for the stepwise development of the Arcellx ARC-T plus sparX cell therapy platform,” according to the press release. The phase 1 trial is currently enrolling patients with relapsed or refractory multiple myeloma to examine the safety and feasibility of the therapy.
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.