Ascentage Pharma Announces Its 9th Orphan Drug Designation From The US FDA In 2020, Setting A Record For Chinese Biopharmaceutical Companies
SUZHOU, China and ROCKVILLE, Md., Jan. 4, 2021 /PRNewswire/ -- Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the US Food and Drug Administration (FDA) has granted an Orphan Drug Designation (ODD) to the company's novel Bcl-2 inhibitor, APG-2575, for the treatment of patients with acute myeloid leukemia (AML). This is the fourth ODD granted to APG-2575 by the US FDA, following the previous ODDs for the treatment of Waldenström macroglobulinemia (WM), chronic lymphocytic leukemia (CLL), and multiple myeloma (MM). To date, four of Ascentage Pharma's investigational drug candidates have been granted a total of nine ODDs by the US FDA, a record number for any Chinese biopharmaceutical company.
AML is a highly heterogenous hematologic malignancy that is more common in the elderly population with a median age at diagnosis of 68 years1. The most recent data from the Surveillance, Epidemiology, and End Results Program (SEER) of the US National Cancer Institute (NCI) estimated 19,940 new cases of AML and 11,180 deaths from this disease in the United States in 2020. Despite the significant advances in therapeutics in recent years, the 5-year survival rate of AML remains at 25%–30%, which still represents a significant unmet medical need for therapies with more durable efficacy and a better safety profile.
The term "orphan drugs" refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the United States, an orphan indication is defined as a disease or condition with a prevalence of less than 200,000 patients in the country. Since the Orphan Drug Act was passed in 1983, the US government has provided incentives and policy support to encourage development of orphan drugs. Therapies granted ODDs by the FDA are qualified for various development incentives, including a tax credit on expenditures incurred in clinical studies, a waiver of the New Drug Application (NDA) fee, possible research grant awarded by the FDA, and most importantly, 7 years of US market exclusivity upon approval.
As a result of the rise of biopharmaceutical innovation in China, a growing number of Chinese biopharmaceutical companies are forging ahead with their global strategies. ODD from the FDA is widely regarded as a key indicator of a company's strength in innovation, and by that, Ascentage Pharma is a clear leader in the field. To date, four of Ascentage Pharma's investigational drug candidates have been granted a total of nine ODDs by the FDA (see table below), a record number for any Chinese biopharmaceutical company so far.