China - CStone Pharmaceuticals ('CStone', HKEX: 2616), a leading biopharmaceutical company focused on the research, development, and commercialization of innovative immuno-oncology therapies and precision medicines, today announced that the AYVAKIT (avapritinib) companion diagnostic (CDx) kit, known as 'Human platelet-derived growth factor receptor alpha (PDGFRA) Gene D842V Mutation Detection Kit', developed in partnership with Genetron Holdings Limited (Genetron Health) has been approved by the China National Medical Products Administration (NMPA).
This CDx kit is the first companion diagnostic product co-developed through the bridging pathway in China to gain regulatory approval following the release of the companion diagnostic guidelines by NMPA. It is used to detect PDGFRA gene mutations in gastrointestinal stromal tumor (GIST) for patients receiving treatment with AYVAKIT.
This approval has made several important breakthroughs. The AYVAKIT CDx kit is the first companion diagnostic developed in China that was approved using a bridging pathway, following the two NMPA guidelines on CDx development ('Guideline for the Registration and Review of Non-original Companion Diagnostic reagent clinical trial for Antitumor Drugs' and 'Guideline for the Registration and Review of Original Companion Diagnostic reagent co-developed with Antitumor Drugs'). Its approval was based on the drug efficacy and tumor testing data obtained from both domestic and global clinical trials of AYVAKIT. Also, it is the first case in which both the drug and CDx have received NMPA Priority Review.
AYVAKIT has demonstrated remarkable efficacy and acceptable safety in patients with unresectable or metastatic GIST harboring PDGFRA exon 18 mutations, including D842V, and it is the first precision medicine approved in China for this specific patient population. The AYVAKIT CDx kit provides a compliant and accurate screening method to identify patients who may benefit from AYVAKIT.
Dr. Archie Tse, Chief Scientific Officer of CStone, said, 'Precision medicine is one of our core strategies at CStone. The approval of the AYVAKIT CDx kit showcases our capability in CDx development for precision medicines in China. This story owes its success to the collaborative efforts among CStone and our experienced diagnostic partner Genetron, as well as Blueprint Medicines. We believe that the approval and commercialization of this CDx kit will help identify more Chinese patients who are candidates for AYVAKIT.'
The approval of the AYVAKIT CDx kit is based on rigorous data that have been shown to address regulatory guidelines. In this regard, Sizhen Wang, Co-founder, Chairman and CEO of Genetron, said, 'With the attitude of being responsible for patients, Genetron and CStone uphold the same strict quality and efficiency standards. Genetron can provide pharmaceutical company customers with efficient companion diagnostic services to help shorten the drug development cycle and promote seamless approval. In the process of working with partners to develop innovative drug and diagnostic products, we hope to make the industry more standardized so that more patients can benefit from precision medicines.'
About AYVAKIT (avapritinib)
AYVAKIT is a precision therapy approved by the National Medical Products Administration (NMPA) of China for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA exon 18 mutation, including PDGFRA D842V mutations. AYVAKIT was approved by the Department of Health (DOH), Hong Kong, China, and Taiwan Food and Drug Administration (TFDA) for the treatment of adults with unresectable or metastatic GIST harboring a PDGFRA D842V mutation.
AYVAKIT is approved by the U.S. Food and Drug Administration (FDA) for the treatment of three indications: adults with indolent systemic mastocytosis (ISM), adults with advanced systemic mastocytosis (advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL), and adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. This medicine is approved in Europe (AYVAKYT) for the treatment of adults with ASM, SM-AHN or MCL, after at least one systemic therapy, and adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
Discovered by CStone's partner Blueprint Medicines, AYVAKIT is a potent, selective and orally available inhibitor of PDGFRA and KIT mutant kinases. CStone has an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of AYVAKIT in Mainland China, Hong Kong, Macau and Taiwan.
About CStone
CStone (HKEX: 2616) is a biopharmaceutical company focused on research, development, and commercialization of innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 14 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, CStone has received eleven NDA approvals for its four drugs. Multiple late-stage drug candidates are now under pivotal clinical trials or registration. CStone's vision is to bring innovative oncology therapies to cancer patients worldwide.
About Genetron Holdings Limited
Genetron Holdings Limited ('Genetron Health' or the 'Company') (Nasdaq: GTH) is a leading precision oncology platform company in China that specializes in cancer molecular profiling and harnesses advanced technologies in molecular biology and data science to transform cancer treatment. The Company has developed a comprehensive oncology portfolio that covers the entire spectrum of cancer management, addressing needs and challenges from early screening, diagnosis and treatment recommendations, as well as continuous disease monitoring and care. Genetron Health also partners with global biopharmaceutical companies and offers customized services and products.
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