According to a story from globenewswire.com, the biopharmaceutical company TG Therapeutics, Inc. recently made an announcement declaring that the US Food and Drug Administration (FDA) had awarded the company Orphan Drug designation. This designation was for TG’s PI3K delta inhibitor therapy umbralisib as a treatment for the rare blood cancer marginal zone lymphoma.
About Marginal Zone Lymphoma (MZL)
Marginal zone B-cell lymphoma, often shortened to marginal zone lymphoma, is a rare type of non-Hodgkin’s lymphoma. This is a slow growing form of lymphoma and they comprise around 12 percent of all non-Hodgkin’s lymphoma cases. As the name implies, this type of lymphoma affects B-cells in the “marginal zones” of different areas of the body. The direct cause of marginal zone lymphoma remains unknown, but a number of risk factors have been identified, such as being more than 60 years old, infectious with the hepatitis C virus, prior history of chronic inflammation or autoimmune conditions, malaria, lupus, and Sjogren syndrome. The symptoms vary depending on the type of marginal zone lymphoma, which are distinguished by the location of malignant activity. They may include lymph node swelling, night sweats, fever, fatigue, weight loss, spleen enlargement, nausea, vomiting, cough, red eyes, shortness of breath, and problems swallowing. Treatment includes chemotherapy, radiation, and immunotherapy. To learn more about marginal zone lymphoma, click here.
About Orphan Drug Designation
Orphan Drug Designation is typically reserved for therapies that are intended to treat a disease that is considered rare. This is defined as any disease that affects less than 200,000 people in the US. To qualify, a therapy must display the potential to offer significant advantages over currently available therapies. Alternatively, it must have the potential to address a medical need that is not being met by current treatment options. 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 is approved.
Umbralisib is currently being tested in a Phase 2 trial as a treatment for marginal zone lymphoma in patients that have already been treated with a single anti-CD20 therapy. Recently announced interim data from this study demonstrated an overall response rate of 52 percent.