FDA Grants Orphan Drug Designation to CARsgen’s CT053 for Multiple Myeloma Treatment

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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CARsgen CT053, multiple myeloma

The U.S. Food and Drug Administration (FDA) has granted the designation of orphan drug to CT053, one of CARsgen Therapeutics’ lead CAR T-cell therapies, for the treatment of patients with multiple myeloma.

Chimeric antigen receptor T-cell therapy, more commonly known as CAR T-cell therapy, is a type of immunotherapy in which a patient’s T-cells — immune cells with anti-cancer activity — are collected and re-engineered in the lab to recognize and eliminate cancer cells. The treated cells are then returned to the patient to fight the tumor.

CARsgen’s CT053 are genetically-modified human T-cells that specifically recognize and eliminate malignant cancer cells containing the B-cell mature antigen (BCMA) — a protein found on the surface of myeloma cells that is considered one of the best targets for this therapy — in patients with multiple myeloma, while leaving healthy cells unharmed.

The Chinese National Medical Products Administration had previously cleared the investigational new drug (IND) application for CT053. Following its IND approval, CT053 started being tested in patients with relapsed or refractory multiple myeloma in clinical trials in China.

“FDA orphan designation is an important regulatory milestone in the continued development and commercialization of CT053 anti-BCMA CAR-T cells,” Zonghai Li, founder, CEO and chief scientific officer of CARsgen, said in a news release.

“CT053 has demonstrated outstanding potency in an exploratory Phase 1 clinical study in China. A total of 19 of 24 patients with relapsed and refractory multiple myeloma showed complete response [cancer eradication]. And importantly, no event of grade 3 [severe] or higher cytokine release syndrome (CRS) was observed,” Li added.

Of note, CRS is a serious side effect that can be triggered by certain medications, in which large amounts of cytokines (molecules that mediate the immune response) are released by modified T-cells, leading to an overactive immune system.

More recently, the FDA has also cleared the IND for CT053, allowing CARsgen to test the treatment in the U.S.

Orphan drug status is given to investigative treatments that aim to help people with a rare disease, which means disorders that affect fewer than 200,000 people in the U.S. The designation comes with certain benefits, including financial incentives for therapy development and commercialization, U.S. market exclusivity for a period of seven years following its approval, FDA support for clinical studies, and special fee exemptions and reductions.