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., January 4, 2021 / PRNewswire / – Ascentage Pharma (6855.HK), a globally focused, clinical-stage biotechnology company engaged in the development of novel therapies for cancers, chronic hepatitis B (CHB) and age-related diseases, today announced that the U.S. Food and The Drug Administration (FDA) has awarded an Orphan Drug Designation (ODD) to the company’s new Bcl-2 inhibitor, APG-2575, for the treatment of patients with acute myeloid leukemia (AML). This is the fourth ODD awarded to APG-2575 by the US FDA, following the previous ODDs for the treatment of Waldenstrom macroglobulinaemia (WM), chronic lymphocytic leukemia (CLL) and multiple myeloma (MM). To date, four of Ascentage Pharma’s investigational drug candidates have received a total of nine ODDs from the US FDA, a record number for any Chinese biopharmaceutical company.

AML is a highly heterogeneous haematological malignancy that is more common in the elderly with a median age at diagnosis of 68 years1. The most recent data from the U.S. National Cancer Institute (NCI) Surveillance, Epidemiology, and End Results Program (SEER) estimated 19,940 new cases of AML and 11,180 deaths from this disease in the United States in 2020. Despite significant advances in therapeutics in recent years, the 5-year survival rate of AML remains at 25% -30%, still representing a significant unmet medical need for therapies with more durable efficacy and 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 Statesan 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 orphan drug development. Therapies awarded by the FDA are eligible for several development incentives, including a tax credit on clinical trial expenses, a New Drug Application (NDA) fee waiver, potential research grant awarded by the FDA, and, more importantly, still, 7 years of US market exclusivity after approval.

As a result of the emergence of biopharmaceutical innovation in Chinaa growing number of Chinese biopharmaceutical companies are pursuing their global strategies. The FDA’s ODD is widely regarded as an important indicator of a company’s innovative strength, making Ascentage Pharma a clear leader in this field. To date, four of Ascentage Pharma’s investigational drug candidates have received a total of nine ODDs from the FDA (see table below), a record number for every Chinese biopharmaceutical company to date.






Chronic Myeloid Leukemia (CML)



Waldenström Macroglobulinaemia (WM)

Chronic Lymphocytic Leukemia (CLL)

Multiple myeloma (MM)

Acute Myeloid Leukemia (AML)



Stomach Cancer (GC)

Acute myeloid leukemia


Sarcoma of the soft tissue


Bcl-2 / Bcl-xL

Small cell lung cancer (SCLC)

According to data published by the US FDA, the proportion of approved orphan drugs has steadily increased over the past 10 years among all approved new therapies. By 2020, new therapies with orphan drug status will account for more than 60 percent of all new drug approvals, with a 10-year high. This shows that the FDA’s support for designated therapies has effectively promoted the development and commercialization of orphan drugs.

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


1. DeSantis CE, Lin CC, Mariotto AB, et al. Cancer treatment and survival 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 the development of novel therapies for cancers, CHB and age-related diseases. On October 28, 2019, Ascentage Pharma is listed on the Main Board of the Stock Exchange of Hong Kong Limited with share code: 6855.HK.

Ascentage Pharma aims to develop therapies that inhibit protein-protein interactions to restore apoptosis or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including new high-potency Bcl-2 and dual Bcl-2 / Bcl-xL inhibitors, as well as candidates targeting 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 major apoptosis regulators. The company conducts more than 40 Phase I / II clinical studies in the US, Australia, and China. HQP1351, the company’s lead drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), has received Orphan Drug Designation (ODD) and Fast Track Designation (FTD) from the U.S. FDA, and a New Drug Application (NDA) for the drug candidate has been submitted 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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SOURCE Ascentage Pharma

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