Investigational Waldenström’s Macroglobulinemia Treatment Earns Orphan Drug Designation

According to a story from BioSpace, the biotechnology company Ascentage Pharma has just announced that it has been given Orphan Drug designation from the US Food and Drug Administration (FDA). This designation is for the company’s experimental drug candidate APG-2575, which is being developed as a therapy for Waldenström’s macroglobulinemia (WM), a rare type of blood cancer. Ascentage focuses on developing treatments for diseases associated with aging, cancer, and chronic hepatitis B.

About Waldenström’s Macroglobulinemia (WM)

Waldenström’s macroglobulinemia, which is also known as lymphoplasmacytic lymphoma, is a rare type of blood cancer that affects two types of B lymphocytes, including plasma cells and lymphoplasmacytoid cells. A distinct characteristic of this type of cancer is the presence of a high concentration of IgM antibodies. It is a slow progressing form of blood cancer, and many patients can lead active lives. While it cannot be cured, it is treatable; some patients are able to experience years of remission without symptoms. There are only about 1,500 new cases per year in the U.S. Although it mostly occurs due to sporadic genetic mutations, a family history increases risk. Symptoms include vision loss, headaches, enlargement of the lymph nodes, liver, and spleen, bleeding nose and gums, weight loss, fatigue, and general weakness. To learn more about WM, click here.

About Orphan Drug Designation

Orphan Drug designation is reserved for medications that are in development to treat diseases and conditions that are considered rare. This is defined as any illness that affects less than 200,000 people in the US. To receive this designation, the drug must meet a medical need that is not satisfied by any current treatment; alternatively, it must display advantages in effectiveness and/or safety over currently available therapies. The designation confers a number of benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the drug gains approval from the FDA.

About APG-2575

APG-2575 is classified as a Bcl-2 inhibitor. It will soon be tested in phase 1b/2 clinical trials both on its own and in combination with ibrutinib and rituximab as a treatment for Waldenström’s macroglobulinemia.


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