Experimental Treatment for Multiple Myeloma Earns Orphan Drug Designation

According to a story from globenewswire.com, the biopharmaceutical company Poseida Therapeutics, Inc. recently announced that the company’s investigational product candidate P-BCMA-101 has been granted Orphan Drug Designation from the US Food and Drug Administration (FDA). This experimental therapy is being developed as a treatment for refractory or relapsed multiple myeloma, a type of rare blood cancer.

About Multiple Myeloma

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 Orphan Drug Designation

Orphan Drug Designation is a special incentive program from the FDA that is specifically meant for therapies developed to treat rare diseases, which is defined as any illness that affects less than 200,000 people in the US. A drug candidate must display potential advantages in effectiveness or safety over currently available treatments. Alternatively, it must satisfy a medical need that is not being met by any current treatments. The designation confers several benefits to the recipient company, such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug gains official approval. 

About P-BCMA-101

P-BCMA-101 is a type of CAR-T cell therapy which is based on a proprietary platform developed by Poseida. This therapy uses longer-lived, stem cell memory T-cells that are self renewing. CAR-T cell therapies involve a process in which T-cells are extracted from a patient. These cells are modified to recognize, target, and destroy cancer cells. After being propagated in the lab setting, these modified T-cells are then reintroduced into the patient’s body. P-BCMA-101 is precisely tuned to target cancer cells that express the B-cell maturation antigen (BCMA).


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