SUZHOU, China, and ROCKVILLE, MD., Nov.22, 2021 — Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announced that the European Commission (EC) has granted the company’s novel drug candidate, olverembatinib (HQP1351), an Orphan Designation for the treatment of chronic myeloid leukemia (CML). This is the first Orphan Designation granted to Ascentage Pharma’s drug candidates in the European Union (EU), and the second granted to olverembatinib globally, following the designation by the US Food and Drug Administration (FDA).
The term “orphan medicines” refers to pharmaceutical products developed for the prevention, diagnosis, and treatment of rare diseases or conditions. In the EU, Orphan Designations are granted by the EC based on the opinions of the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), and only those therapies treating life-threatening or chronically debilitating diseases or conditions affecting less than 5 in 10,000 people in the EU and represent huge unmet medical needs are granted the Orphan Designation. Moreover, designated drugs must be able to demonstrate through non-clinical and clinical data that it can potentially provide greater therapeutic benefit than existing therapies. The EMA offers a range of incentives to encourage the development of designated orphan medicines. This Orphan Designation for olverembatinib qualifies the drug candidate for great regulatory supports in the subsequent clinical development and commercialization in the European Union, including protocol assistance, fee reductions, and most importantly, 10 years of market exclusivity upon approval.
CML is a rare hematologic malignancy that has an annual incidence of 2.43 per 10,000 in the 27 member nations of the EU. BCR-ABL tyrosine kinase inhibitors (TKIs) have significantly improved the clinical management of CML. However, despite clinical benefits offered by the first- and second-generation TKIs, many patients develop drug resistance. Such acquired resistance to TKIs is a major challenge in the treatment of CML. BCR-ABL kinase mutations represent a key mechanism of acquired drug resistance; T315I, which is the most- common drug-resistant mutation, occurs in about 25% of patients with drug-resistant CML. Patients with T315I-mutant CML are resistant to both first- and second-generation BCR-ABL inhibitors, hence presenting an urgent and high unmet medical need for third-generation BCR-ABL inhibitors to more effectively target the T315I mutation.
Olverembatinib is a novel third-generation BCR-ABL TKI developed by Ascentage Pharma for the treatment of patients with CML resistant to first- and second-generation TKIs, including those harboring the T315I mutation. The New Drug Application (NDA) for olverembatinib for the treatment of patients with chronic phase CML (CML-CP) or accelerated-phase CML (CML-AP) harboring the T315I mutation is currently under review in China. If approved by the National Medical Products Administration (NMPA), olverembatinib would become the first approved third-generation BCR-ABL TKI in China and the second globally. Previously, olverembatinib was granted a Breakthrough Therapy Designation by the Center for Drug Evaluation (CDE) of China NMPA for the treatment of patients with CML-CP resistant and/or intolerant to first- and second-generation TKIs. In the United States, olverembatinib was granted an Orphan Drug Designation and Fast Track Designation by the US FDA. Furthermore, since 2018, the clinical data from olverembatinib studies have been selected for oral presentations at the American Society of Hematology (ASH) Annual Meetings for four consecutive years, and was nominated for “Best of ASH” in 2019.
“There is enormous unmet medical need in the treatment of CML globally. This is why olverembatinib has received widespread interest from the international hematology community since the first publication of its clinical data,” said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. “This Orphan Designation by the EC indicates the recognition of olverembatinib’s potential in the management of CML, marking yet another important milestone in the development and commercialization of this drug candidate. As the first Orphan Designation granted to Ascentage Pharma’s drug candidates by the EC, it is also a testament to the company’s deepening global expansion. Leveraging the policy support from the EC, we will continue to expedite the global clinical development and commercialization of olverembatinib. We are hopeful that these efforts will allow us to bring this China-developed potential novel therapeutic to patients around the world as soon as possible.”
About Ascentage Pharma
Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, 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, Europe, and China. Ascentage Pharma has been designated for multiple Major National R&D Projects, including five Major New Drug Projects, one New Drug Incubator status, four Innovative Drug Programs, and one Major Project for the Prevention and Treatment of Infectious Diseases. Ascentage Pharma has already submitted a New Drug Application (NDA) for olverembatinib, the company’s core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), and was subsequently granted Priority Review status and a Breakthrough Therapy for the drug candidate. In addition, the olverembatinib was also granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA. To date, Ascentage Pharma has obtained a total of 12 ODDs from the US FDA and 1 Orphan Designation from the EC for four of the company’s investigational drug candidates.
Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, MSD, and AstraZeneca. The company has built a talented team with global experience in the discovery and development of innovative drugs and is setting up its world-class commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients.
The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, Ascentage Pharma undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events, or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.
 GBD 2019 Diseases and Injuries Collaborators Supplementary