CAR-T Cell Therapy Earns Regenerative Medicine Advanced Therapy Designation for Multiple Myeloma

According to a story from Hematology Times, an experimental CAR-T cell therapy called P-BCMA-101 was recently granted regenerative medicine advanced therapy (RMAT) designation from the US Food and Drug Administration. This designation was given to the drug as a treatment for relapsed or refractory multiple myeloma, a cancer which affects plasma cells. 

Multiple myeloma, which is occasionally referred to as plasma cell myeloma, is a blood cancer that affects plasma cells. These are white blood cells that produce antibodies. The overall cause of multiple myeloma is not well understood, however, some risk factors have been identified. These include obesity, family history, smoldering myeloma, and monoclonal gammopathy of undetermined significance. These last two conditions have the potential to develop into multiple myeloma. Symptoms of this cancer include bone pain, infections, anemia, kidney failure, overly thick blood, confusion, fatigue, headaches, and amyloidosis. Treatment includes chemo, stem cell transplant, and other medications for relapsed disease, which is common. Five year survival rate is 49 percent in the US. To learn more about multiple myeloma, click here.

About Regenerative Medicine Advanced Therapy Designation (RMAT)

RMAT designation is intended to speed up the development process for advanced, regenerative therapies that display the potential to treat, reverse, cure, or otherwise modify a serious, life threatening disease or condition. The designation is typically reserved for therapies that display the potential to address currently unmet medical needs in preliminary data.

RMAT designation combines the benefits of both Breakthrough Therapy designation and Fast Track designation. These benefits include close collaboration with the FDA to facilitate the development of the drug and support meaningful data collection, eligibility for Accelerated Approval and Priority Review, and Rolling Review, which allows the company to submit portions of its New Drug Application (NDA) as they are completed rather than being forced to wait until the entire application has been filled. The FDA offers more direct guidance for development and commits more strongly to the approval of the drug.

About CAR-T Cell Therapy

CAR-T cell therapies like P-BCMA-101 work by extracting T cells from the patient, modifying them to recognize a target found on cancer cells, proliferating these modified cells in a lab, and then reintroducing them into the body of the patient. These modified cells are capable of identifying and destroying cancer cells.

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