CStone Pharmaceuticals (HKG: 2616), a China-based biopharmaceutical company, has announced that it has received marketing approval from the National Medical Products Administration (NMPA) for its “Human PDGFRA Gene D842V Mutation Detection Kit”. This companion diagnostic (CDx) kit, co-developed with Beijing-based Genetron Holdings Ltd (Nasdaq: GTH), is used for the detection of PDGFRA gene mutations in patients with gastrointestinal stromal tumors (GIST), marking it as the first CDx kit developed through the bridging study channel.
Significance of the Approval
The approval of this kit is a significant milestone, as it is also the first home-based CDx kit to be approved with both domestic and overseas effectiveness data. This recognition underestimates the importance of precision medicine in the diagnosis and treatment of GIST, a rare form of cancer.
Ayvakit: A Targeted TKI for GIST and Mastocytosis
Ayvakit, a KIT and PDGFRA-targeted tyrosine kinase inhibitor (TKI), was in-licensed from the US firm Blueprint Medicine in June 2018. The drug has been approved to treat a range of conditions in the Chinese mainland, Taiwan, and Hong Kong, including unresectable or metastatic GIST with PDGFRA exon 18 mutation (including PDGFRA D842V mutation) and advanced systemic mastocytosis (SM). In Europe, Ayvakit is approved for gastrointestinal stromal tumors with PDGFRA D842V mutation that cannot be resected or metastasized, as well as advanced systemic mastocytosis (ASM), SM with blood tumor (SM-AHN), and mast cell leukemia (MCL) after at least one systemic treatment.
Rare Disease Therapy Status
Ayvakit holds a rare disease therapy status, as GIST was included in the second batch of the rare disease list. This designation highlights the importance of developing and approving therapies for rare diseases, which can significantly improve the lives of patients with these conditions.-Fineline Info & Tech
