Experimental Therapies for Small Cell Lung Cancer and Acute Myeloid Leukemia Earn Orphan Drug Designation

According to a story from BioSpace, the biotechnology company Ascentage Pharma announced that two of its investigational treatments have recently received Orphan Drug designation from the US Food and Drug Administration (FDA). These treatments include APG-115, which is being developed to treat acute myeloid leukemia; and APG-1252, which is being developed as a treatment for small cell lung cancer. Ascsentage prioritizes developing treatments for age-related diseases, cancer, and chronic hepatitis B.

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia, also known as acute myelogenous leukemia, is a type of blood cancer which affects myeloblasts, stem cells that would normally develop into myeloid white blood cells. Symptoms include an increased risk of infection, easy bruising and bleeding, fatigue, shortness of breath, fever, weight and appetite loss, anemia, and bone/joint pain. Treatment for this cancer is most often chemotherapy or stem cell transplant; there are very limited options for patients with relapsed disease. To learn more about acute myeloid leukemia, click here.

About Small Cell Lung Cancer

Small cell lung cancer is an aggressive type of lung cancer that is responsible for only about 10 to 15 percent of lung cancer cases. Lung cancer as a whole is not considered rare, but small cell lung cancer is. Symptoms of this cancer include chest pain, coughing up blood, weight loss, and shortness of breath. This cancer metastasizes rapidly and most patients are diagnosed when this process has already begun. This cancer relapses easily and the five year survival rate is meager at just 20 percent and is even lower for metastatic disease. To learn more about small cell lung cancer, click here.

About Orphan Drug Designation

Orphan Drug designation is reserved for therapies that are in development to treat diseases that are considered rare, which is defined as any illness that affects less than 200,000 people in the US. The treatment must display potential advantages in safety or effectiveness over current treatments in order to qualify. Alternatively, it must have the potential to satisfy an unmet medical need. Recipients of this designation receive several benefits, such as the waiving of certain fees, tax breaks, and a period of market exclusivity lasting seven years if the drug gets FDA approval. 

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