RARE Daily

FDA Grants RMAT and Fast Track Designations to Iaso Bio CT103A for Rare Blood Cancer

February 13, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has granted both Regenerative Medicine Advanced Therapy designation and Fast Track designation to Iaso Biotherapeutics’ investigational new drug BCMA CAR-T CT103A for relapsed/refractory multiple myeloma.

Multiple myeloma is a rare cancer characterized by excessive production and improper function of plasma cells, which are a type of white blood cell and found in the bone marrow. Excessive plasma cells may eventually mass together to form a tumor or tumors in various sites of the body, especially the bone marrow. When multiple tumors are present or the bone marrow has greater than 10 percent plasma cells, the term multiple myeloma is used. Major symptoms of multiple myeloma may include bone pain, especially in the back and the ribs; low levels of circulating red blood cells resulting in weakness and fatigue, and renal abnormalities. The cause of multiple myeloma is unknown.

Iaso’s CT103A is a BCMA chimeric antigen receptor autologous T cell injection, a lentiviral vector containing a CAR structure with a fully human scFv, CD8a hinger and transmembrane, 4-1BB co-stimulatory and CD3ζ activation domains. Based on strict selection and screening, utilizing a proprietary in-house optimization platform, and integrated in-house manufacturing process improvement, Iaso says the construct of CT103A is potent and shows prolonged persistency in patients.

Established under the 21st Century Cures Act, Regenerative Medicine Advanced Therapy (RMAT) designation is intended to help the FDA facilitate an efficient development program of any drug that qualifies as RMAT, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products. It is intended to treat, modify, reverse or cure a serious or life threatening disease or condition; and has preliminary clinical evidence to indicated the drug has the potential to address unmet medical needs for such a disease or condition.

Fast Track designation is designed to accelerate the development and review of treatments for serious and life-threatening diseases where no treatment exists or where the treatment in discovery may be better than what is currently available, thus enabling drugs to potentially reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.

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