On January 28, 2021 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, reported 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 follicular lymphoma (FL) (Press release, Ascentage Pharma, JAN 28, 2021, View Source [SID1234574432]). This is the fifth ODD granted to the company’s core asset APG-2575 by the US FDA, following the previous ODDs for the treatment of Waldenström macroglobulinemia (WM), chronic lymphocytic leukemia (CLL), multiple myeloma (MM), and acute myeloid leukemia (AML). To date, four of Ascentage Pharma’s investigational drug candidates have been granted a total of ten ODDs by the US FDA, a record number for any Chinese biopharmaceutical company.

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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.

FL is a heterogeneous disease and the second most common subtype of non-Hodgkin lymphoma (NHL) among adult patients in the US, accounting for about 20% of all NHL incidences[1]. Defined as a proliferation of malignant germinal center B cells, FL is also the most common subtype of indolent B-cell lymphoma[1],[2]. Although currently available therapies have significantly improved the overall survival of patients with FL, the disease still remains incurable[2],[3]. Majority of patients with FL relapse or experience disease progression after receiving the initial therapy, and experience multiple relapses thereafter requiring subsequent lines of therapy[4]. Despite the multiple treatment options for newly diagnosed and relapsed/refractory FL (r/r FL), FL still represents an unmet medical need, especially in those patients who are at high risk, relapsed early, have histological transformation, relapsed from available treatment options, or developed intolerance to available therapies.

APG-2575 is a novel, orally administered small-molecule Bcl-2‒selective inhibitor being developed by Ascentage Pharma. APG-2575 is designed to treat hematologic malignancies and solid tumors by selectively blocking antiapoptotic protein Bcl-2 to restore the normal apoptosis process in cancer cells. APG-2575 is the first China-developed Bcl-2 inhibitor entering clinical development in China. Previously, APG-2575 had received clearances and approvals for multiple Phase Ib/II clinical studies in China, Australia, and the US, and is currently being developed in a range of hematologic malignancies globally.

"This ODD from the FDA is the fifth one granted to APG-2575, a key drug candidate in our apoptosis-targeting pipeline, also the tenth obtained by the company. This latest designation enhances our leadership position in terms of the number of ODDs obtained by any Chinese biopharmaceutical company, further demonstrating our capabilities in innovation globally," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "We hope the FDA’s policy supports for orphan drug development will enable us to further accelerate the clinical development of APG-2575 and other ODD designated drug candidates in our pipeline, which hopefully can soon bring novel therapeutics to patients in need."