CORXEL Pharmaceuticals, a portfolio company of RTW Biotech Opportunities, has achieved a significant regulatory milestone with the acceptance of its new drug application by China's National Medical Products Administration (NMPA) for aficamten. The application seeks approval for aficamten in the treatment of obstructive hypertrophic cardiomyopathy (HCM).
Aficamten is described by RTW as an "investigational, next-in-class selective small molecule cardiac myosin inhibitor." This drug has already received Breakthrough Therapy Designation from both the US Food & Drug Administration (FDA) and the NMPA for symptomatic obstructive HCM.
Understanding Hypertrophic Cardiomyopathy
HCM is a genetic heart disease characterized by abnormal thickening of the heart muscle, which ultimately impairs the heart's pumping function. It is the most common inherited cardiovascular disorder. According to RTW's announcement, approximately 132,000 to 330,000 patients have been diagnosed with HCM in China. However, the estimated number of undiagnosed individuals ranges from 748,000 to 1,870,000, highlighting a significant unmet medical need.
CORXEL's Role and Collaboration
CORXEL, formerly known as Ji Xing Pharmaceuticals, is a biotech company focused on developing therapies for underserved patients with cardiometabolic diseases. Founded and launched by RTW Investments in 2019, CORXEL entered into a license and collaboration agreement with Cytokinetics in July 2020. This agreement grants CORXEL an exclusive license to develop and commercialize aficamten in Greater China.
