According to a story from globenewswire.com, the pharmaceutical company Portola Pharmaceuticals recently announced that its investigational product cerdulatinib has earned Orphan Drug designation from the US Food and Drug Administration for the treatment of peripheral T-cell lymphoma, a rare and aggressive form of Non-Hodgkin lymphoma. There are currently significant limitations to the treatment approaches available for this form of lymphoma.
About Peripheral T-Cell Lymphoma
Peripheral T-cell lymphoma is a type of rare blood cancer that, as the name implies, primarily affects T-cells, a type of white blood cell. It can also affect NK cells (“natural killer” cells). Only about 10 to 15 percent of non-Hodgkin lymphoma cases are of this type. It is also rarer in the US and is more common in Asia, Africa or the Caribbean. The cause of peripheral T-cell lymphoma is poorly understood, but exposure to the Epstein-Barr virus and the human T-cell leukemia virus 1 are both risk factors for this cancer. Symptoms of this disease vary depending on the sub-variant, but may include weight loss, fatigue, painless swelling of the lymph nodes, rash, and night sweats. Treatment may include chemo, targeted therapies, and stem cell transplant. No standard of care has been established for relapsed disease. To learn more about peripheral T-cell lymphoma, click here.
Orphan Drug Designation
Orphan Drug designation confers several benefits to the company responsible for developing the therapy that is granted it. This designation is reserved for treatments that are intended to treat diseases that are considered rare, which is defined as those that affect 200,000 people or less in a given year. In addition, the drug must either fulfill a currently unmet medical need or display significant advantages in effectiveness or safety in comparison to current treatments. Benefits of the designation include tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug is successfully approved.
Cerdulatinib is a spleen tyrosine kinase (SyK) and janus kinase (JAK) that has the potential to be effective in treating a diverse array of different types of Non-Hodgkin lymphoma. In addition to peripheral T-cell lymphoma, cerdulatinib could also be of potential use against small lymphocytic lymphoma and relapsed follicular lymphoma.