MB-106 being developed by Mustang Biosciences has been given an orphan drug designation by The United States Food and Drug Administration (FDA) for use in patients with Waldenstrom macroglobulinemia, an extremely rare type of blood cancer.
The Orphan Drug Act was created to incentivize pharmaceutical companies to develop drugs to treat patients with rare diseases. When a drug is granted an orphan drug designation, the pharmaceutical company developing the drug is eligible for certain incentives including tax credits for qualified clinical trials.
Waldenström’s macroglobulinemia is a rare type of B-cell lymphoma that gradually progresses and causes B lymphocytes to grow within the bone marrow, lymph nodes, liver, and spleen. The abnormal B cells also overproduce a protein known as immunoglobulin M or IgM that may lead to excessive bleeding and problems with vision and the nervous system.
The FDA has already approved several chimeric antigen receptor (CAR) technologies for the treatment of malignant lymphoma. The different agents utilize slightly different methods of genetic engineering to transform a patient’s T cells into CAR-T cells.
CAR therapies utilize T-cells (CART T), a patient’s own immune cells that are re-programmed to recognize and kill cancer cells throughout the body. The process involves the removal of some T cells from a patient, and through laboratory processes, these T cells are re-programmed to identify a patient’s cancer cells. Once the T cells have been programmed to identify a patient’s cancer cells, they are replicated in the laboratory, and infused back into the patient. These re-programmed T cells circulate throughout the body, identifying the cancer cells and mounting an immune attack against them. Simultaneously, the T cells are replicating within the body, so that more of the immune cells can identify and attack the cancer cells.
MB-106 is a CD20-targeted autologous CAR-T cell therapy currently being evaluated in patients with various high-risk B-cell non-Hodgkin lymphomas. Findings from a clinical trial have shown that treatment was and safe and effective in patients with other advanced lymphoma.1
Reference:
1. https://www.mustangbio.com/wp-content/uploads/2020/12/CD20-CART-ASH2020-FINAL.pdf
