On February 16, 2022, Healio shared that CT103A, a therapy for adult patients with relapsed or refractory (R/R) multiple myeloma (MM), had received Orphan Drug designation from the United States Food and Drug Administration (FDA). This status is given to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, a rare condition is any affecting under 200,000 Americans. As a benefit, the drug developer(s) – in this case, IASO Biotherapeutics and Innovent Biologics – receive multiple incentives, including fee waivers, tax credits, increased regulatory assistance, and 7 years market exclusivity upon approval.

About CT103A

According to the Multiple Myeloma Hub, CT103A is:

A chimeric antigen receptor (CAR) T-cell therapy that targets B-cell maturation antigen (BCMA) which is found on the surface of myeloma plasma cells. It contains a fully-human BCMA antibody, designed to reduce the toxic side effects often induced by non-human constructs.

Currently, researchers are evaluating CT103A in a Phase 1/2 FUMANBA-1 clinical trial. Enrolled participants have been diagnosed with R/R multiple myeloma and have previously been treated with at least 3 other forms of treatment. Preliminary data suggests that CT103A had very strong overall and complete response rates. While the treatment was relatively safe, over 90% of those involved experienced cytokine release syndrome, an acute and systemic inflammatory response. Learn more about cytokine release syndrome. 

What is Multiple Myeloma (MM)?

Multiple myeloma (MM) is a rare cancer which forms in plasma cells, a type of white blood cell which normally plays a role in immune response and fighting infections. While doctors are not sure exactly what causes multiple myeloma, some research has shown that many patients lack part or all of chromosome 13. This cancer produces abnormal cells, which crowd healthy blood cells out of the bone marrow, as well as abnormal M proteins. Risk factors include being male, being Black, increasing age, or having a family history of multiple myeloma. Symptoms and characteristics associated with multiple myeloma include:

• Unintended weight loss
• Bone pain, especially in the spine or chest
• Anemia (low red blood cell count) 
• Nausea
• Fatigue
• Blood clots
• Easy bruising and bleeding
• Frequent or recurrent infections
• Osteoporosis and increased risk of bone fractures
• Appetite loss
• Constipation
• Reduced kidney function
• Mental fogginess or confusion
• Excessive thirst
• Hypercalcemia (high blood calcium levels)
• Weakness or numbness in the legs