In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, “rare” is defined as affecting 200,000 Americans or less. Once a drug receives this status, the drug developer also receives benefits such as fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon approval. According to Myeloma Research News, the FDA recently granted Orphan Drug status to GC012F, an experimental CAR T-cell therapy for multiple myeloma (MM).
GC012F
Developed by Gracell Biotechnologies, GC012F is described as:
an autologous dual CAR-T targeted BCMA and CD19.
This therapy, administered as an injection, uses modified immune T-cells from the patient’s own body. Multiple myeloma cells have express high levels of B-cell maturation antigen (BCMA) on their surface. CD19 plays a role in developing new cancerous cells. Through this treatment, the T-cells are modified to recognize and attack cells with BCMA or CD19. Using the proprietary FasTCAR technology, Gracell Biotechnologies is also able to manufacture this treatment quickly — within 1-2 days. Researchers and scientists believe this could help improve the quickness of treatment as well as the efficacy and durability.
Currently, researchers are evaluating GC012F in three separate studies, which include high-risk patients. So far, preliminary data highlights a high overall or partial response rate to treatment. Those treated with GC012F saw tumor cells removed from circulation and did not experience cytokine release syndrome, which can be an issue with other immunotherapy treatments. Additionally, GC012F showed a durable and sustained treatment response, with over 50% of those treated still responding over 1 year later.
Multiple Myeloma (MM)
Multiple myeloma (MM) is a rare cancer which forms in plasma cells, a type of white blood cell that usually plays a role in antibody creation and immune response to infections. Doctors are not sure exactly what causes multiple myeloma. However, some myeloma cells are missing all or part of chromosome 13, suggesting a potential underlying genetic cause. As abnormal and cancerous cells build-up in bone marrow, healthy blood cells are crowded out, causing a variety of health issues. Symptoms of multiple myeloma include:
- Nausea
- Bone pain which is particularly prevalent in the chest or spine
- Appetite loss
- Fever
- Pathological fractures
- Constipation
- Fatigue
- Unintended weight loss
- Frequent infections
- Numbness or weakness, particularly in the legs
- Excessive thirst
- Mental fogginess/confusion
- Hypercalcemia