On November 25, Olverembatinib Tablets (trade name: Nileck®), a Class 1 New Drug independently developed by the Guangzhou Institute of Biomedicine and Health (GIBH) under the Chinese Academy of Sciences, officially obtained marketing approval from the National Medical Products Administration (NMPA). It is indicated for the treatment of adult patients with chronic phase (CP) or accelerated phase (AP) chronic myeloid leukemia (CML) harboring the T315I mutation, confirmed by a fully validated detection method, who are resistant to any tyrosine kinase inhibitor (TKI). This marks the first approval of a Class 1.1 New Drug since the establishment of GIBH.
CML is a hematologic malignancy characterized by abnormal proliferation of leukocytes. The launch of BCR-ABL-targeted TKIs has revolutionized the treatment landscape of CML. However, acquired resistance remains a major challenge in CML management. BCR-ABL kinase domain mutations are one of the key mechanisms underlying acquired resistance, among which the T315I mutation is a common type, accounting for approximately 25% of cases in drug-resistant CML. Patients with CML harboring the T315I mutation are resistant to all currently available first and second-generation BCR-ABL inhibitors, leaving them with no effective treatment options in the past.
In 2006, GIBH recruited the research team led by Professor Ding Ke, and in 2008, initiated the layout of novel drug development for malignant tumor treatment based on tyrosine kinase inhibitors. In 2013, preliminary laboratory validation identified D824 as a promising anti-leukemia candidate drug, and the related patents were transferred to Guangzhou Shunjian Biopharmaceutical Technology Co., Ltd. for industrialization. In 2016, D824 (HQP1351, Olverembatinib) was granted clinical trial approval as a Class 1.1 New Drug, with subsequent clinical trials advanced by Jiangsu Yasen Pharmaceutical Co., Ltd., the parent company of Guangzhou Shunjian. On November 24, 2021, Olverembatinib received marketing approval, culminating a 13-year R&D journey. This stands as a typical successful case of achievement transformation in new drug research and development, featuring R&D by an academic institution followed by industrialization led by pharmaceutical enterprises.
Olverembatinib is a potential best-in-class novel drug and a supported product under the National Major Scientific and Technological Special Project for "Significant New Drug Development". The approval of Nileck® is primarily based on clinical data from two pivotal Phase II registration studies, namely the HQP1351CC201 study and the HQP1351CC202 study. Clinical data demonstrate that Nileck® exhibits favorable efficacy and tolerability in TKI-resistant CML-CP and CML-AP patients harboring the T315I mutation, with remission rates and depth of remission further improving as the treatment duration extends.
As China's first third-generation BCR-ABL targeted therapy for drug-resistant CML, the approval of Olverembatinib will break the treatment bottleneck for Chinese patients with T315I mutation-driven drug resistance and address the predicament of having no available treatment options.
