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[1] 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 th...
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