Shanghai-based Corxel Pharmaceuticals Co. Ltd., an RTW Biotech Opportunities portfolio company, has announced that the China National Medical Products Administration (NMPA) has accepted its new drug application (NDA) for Aficamten. This drug is intended for the treatment of obstructive hypertrophic cardiomyopathy (oHCM), a genetic heart condition characterized by abnormal thickening of the heart muscle, which impairs the heart's ability to pump blood effectively.
Addressing a Significant Unmet Need
Obstructive hypertrophic cardiomyopathy affects an estimated 130,000 to 330,000 patients in China. The condition leads to symptoms such as shortness of breath, chest pain, and fatigue, significantly impacting the quality of life. Current treatment options are limited, often focusing on symptom management rather than addressing the underlying cause of the disease. Aficamten represents a potential advancement by targeting the molecular mechanisms driving myocardial hypercontractility and hypertrophy.
Aficamten's Mechanism and Regulatory Status
Aficamten is a selective cardiac myosin inhibitor designed to reduce the excessive contractility of the heart muscle in oHCM patients. By modulating the number of active myosin-actin cross-bridges, Aficamten aims to improve cardiac function and reduce symptoms. The acceptance of the NDA by the NMPA marks a significant step toward bringing this novel therapy to patients in China.
Notably, Aficamten has also received breakthrough therapy designation from the U.S. Food and Drug Administration (FDA). This designation is granted to drugs intended to treat a serious condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies.
