Partners CStone Pharmaceuticals (HKG: 2616) and Blueprint Medicines have announced that the US FDA has approved Ayvakit (avapritinib), a tyrosine kinase inhibitor, for the treatment of adults with indolent systemic mastocytosis (ISM). ISM is a rare hematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems, significantly impacting patients’ quality of life. The regulatory decision is based on the positive results of the PIONEER study, which showed significant clinical benefits for ISM patients who received 48 weeks of treatment with Ayvakit. Additionally, Blueprint Medicines has submitted a market filing with the European Medicine Agency for the same indication.
Ayvakit: A Targeted Therapy for Rare Disorders
Ayvakit is a KIT and PDGFRA-targeted tyrosine kinase inhibitor (TKI) licensed from Blueprint Medicine in June 2018. The drug has been approved to treat a variety of conditions, including unresectable or metastatic gastrointestinal stromal tumors (GIST) with PDGFRA exon 18 mutation (including PDGFRA D842V mutation) and advanced systemic mastocytosis (SM) in the Chinese mainland, Taiwan, and Hong Kong. In Europe, Ayvakit is approved for advanced systemic mastocytosis (ASM), SM with blood tumor (SM-AHN), and mast cell leukemia (MCL) after at least one systemic treatment, as well as gastrointestinal stromal tumors with PDGFRA D842V mutation that cannot be resected or metastasized.
Expanding Access to Life-Changing Treatments
The approval of Ayvakit for ISM in the US marks a significant milestone in expanding access to life-changing treatments for patients suffering from this rare and debilitating disorder. With its proven efficacy and safety profile, Ayvakit offers hope for improved outcomes and quality of life for those affected by ISM and other related conditions.-Fineline Info & Tech
