Shanghai-based Corxel Pharmaceuticals Co. Ltd., formerly Ji Xing Pharmaceuticals Ltd, has achieved a significant milestone with the acceptance of its new drug application (NDA) for Aficamten by China's National Medical Products Administration (NMPA). Aficamten is being developed as a treatment for obstructive hypertrophic cardiomyopathy (HCM), a genetic heart condition characterized by abnormal thickening of the heart muscle, limiting its pumping function.
Obstructive Hypertrophic Cardiomyopathy (HCM)
Obstructive HCM affects an estimated 130,000 to 330,000 patients in China. The condition impairs the heart's ability to pump blood effectively, leading to symptoms such as shortness of breath, chest pain, and fatigue. Current treatment options are limited, highlighting the unmet medical need for more effective therapies.
Aficamten: A Potential Breakthrough
Aficamten has also received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA), underscoring its potential to address a critical gap in the treatment of obstructive HCM. The drug's mechanism of action targets the underlying cause of the disease, offering a potential improvement over existing symptomatic treatments.
Corxel Pharmaceuticals
Corxel Pharmaceuticals was founded and launched by RTW Investments in 2019, with the specific aim of developing and commercializing innovative therapies like Aficamten. The acceptance of the NDA in China represents a major step forward for the company and for patients suffering from obstructive HCM.
