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. 5, 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, 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 years[1]. 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.






Chronic Myeloid Leukemia (CML)



Waldenström Macroglobulinemia (WM)

Chronic Lymphocytic Leukemia (CLL)

Multiple Myeloma (MM)

Acute Myeloid Leukemia (AML)



Gastric Cancer (GC)

Acute Myeloid Leukemia


Soft Tissue Sarcoma



Small Cell Lung Cancer (SCLC)

According to data published by the US FDA, there has been a steady rise in the proportion of approved orphan drugs among all approved novel therapies in the in the past 10 years. In 2020, novel therapies with orphan drug status account for over 60 percent of all new drug approvals, hitting a 10-year high. This shows the FDA’s support to designated therapies has effectively fostered the development and commercialization of orphan drugs.

“Receiving nine ODDs from the FDA in just a year is record-setting, by speed and number, among Chinese biopharmaceutical companies; it is also an impressive achievement even by global standard,” said Dr. Dajun Yang, Chairman & CEO of Ascentage Pharma. “This accomplishment is a result of our unwavering commitment to our founding mission of ‘addressing unmet medical needs in China and around the world’. I hope that with the various policy supports for ODD designated therapies, we will be able to accelerate the clinical development of our drug candidates, which hopefully can soon benefit patients in need.”


[1]. DeSantis CE, Lin CC, Mariotto AB, et al. Cancer Treatment and Survivorship Statistics, 2014. CA Cancer J Clin 2014;64:252-271.

About Ascentage Pharma

Ascentage Pharma (6855.HK) is a globally focused, clinical-stage biotechnology company engaged in developing novel therapies for cancers, CHB, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 6855.HK.

Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors (TKIs). Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 40 Phase I/II clinical trials in the US, Australia, and China. HQP1351, the company’s core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), has been granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and a New Drug Application (NDA) for the drug candidate has been submitted in China. To date, Ascentage Pharma has obtained a total of nine ODDs from the US FDA for four of the company’s investigational drug candidates.

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