AYVAKIT ® (avapritinib) is China’s first approved precision therapy for patients with PDGFRA Exon 18 Mutant Gastrointestinal Stromal Tumor discovered by CStone’s partner Blueprint Medicines.
AYVAKIT (avapritinib) is a kinase inhibitor for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA exon 18 mutation.
The U.S. Food and Drug Administration (FDA) has approved AYVAKITTM for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. This medicine is approved by the European Commission under the brand name AYVAKYT® for the treatment of adults with unresectable or metastatic GIST harboring the PDGFRA D842V mutation.
AYVAKIT/AYVAKYT is not approved for the treatment of any other indication in China by the NMPA, in the U.S. by the FDA or in Europe by the European Commission, or for any indication in any other jurisdiction by any other health authority.
CStone and Blueprint Medicines have an exclusive collaboration and license agreement for the development and commercialization of avapritinib and certain other drug candidates in Mainland China, Hong Kong, Macau and Taiwan. Blueprint Medicines retains development and commercial rights for avapritinib in the rest of the world.
Blueprint Medicines is developing avapritinib globally for the treatment of advanced and indolent systemic mastocytosis (SM). The FDA granted breakthrough therapy designation to avapritinib for the treatment of advanced SM, including the subtypes of aggressive SM, SM with an associated hematologic neoplasm and mast cell leukemia, and for the treatment of moderate to severe indolent SM.
GAVRETO® (pralsetinib) is China's first approved selective RET inhibitor discovered by CStone’s partner Blueprint Medicines. CStone has an exclusive collaboration and license agreement with Blueprint Medicines for the development and commercialization of GAVRETO in Greater China, which encompasses Mainland China, Hong Kong, Macau and Taiwan.
GAVRETO is a once-daily oral targeted therapy for the treatment of adults with locally advanced or metastatic RET fusion-positive NSCLC after platinum-based chemotherapy.
GAVRETO has been approved by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with metastatic RET fusion-positive NSCLC as detected by an FDA approved test, adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC), and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).
GAVRETO is not approved for the treatment of any other indication in China by the NMPA or in the U.S. by the FDA, or for any indication in any other jurisdiction by any other health authority.
GAVRETO is designed to selectively and potently target oncogenic RET alterations, including secondary RET mutations predicted to drive resistance to treatment. In preclinical studies, GAVRETO inhibited RET at lower concentrations than other pharmacologically relevant kinases, including VEGFR2, FGFR2, and JAK2.
The European Medicines Agency validated a marketing authorization application for GAVRETO for the treatment of RET fusion-positive NSCLC, and the review is ongoing. The FDA granted breakthrough therapy designation to GAVRETO for the treatment of RET fusion-positive NSCLC that has progressed following platinum-based chemotherapy and for RET mutation-positive MTC that requires systemic treatment and for which there are no alternative treatments.
TIBSOVO® is an oral targeted IDH1 inhibitor. The NMPA of China has approved the NDA of TIBSOVO® for the treatment of adult patients with relapsed/refractory acute myeloid leukemia who have a susceptible IDH1 mutation.
In China, TIBSOVO was selected in the list of the third batch of Overseas New Drugs Urgently Needed in Clinical Settings by the Center for Drug Evaluation, NMPA in China, and granted fast-track designation. As a potent and highly selective first-in-class oral IDH1 inhibitor, TIBSOVO was also recommended by the 2020 edition of the CSCO Guidelines for Diagnosis and Treatment of Hematological Malignancies due to its proven clinical advantages.
TIBSOVO® is currently approved in the U.S. as monotherapy for the treatment for the treatment of adults with IDH1-mutant relapsed or refractory acute myeloid leukemia (AML), and for adults with newly-diagnosed AML with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in adult patients who are ≥ 75 years old or who have comorbidities that preclude use of intensive induction chemotherapy. In 2021, TIBSOVO® was the first and only targeted therapy approved for patients with previously treated locally advanced or metastatic cholangiocarcinoma with an IDH1-mutation as detected by an FDA-approved test.
The U.S. FDA has granted Breakthrough Therapy Designation for TIBSOVO in combination with azacitidine for this indication and Breakthrough Therapy Designation for TIBSOVO for the treatment of adult patients with relapsed or refractory myelodysplastic syndrome (MDS) with a susceptible IDH1 mutation.