FDA Accepts Cytokinetics' NDA for Aficamten to Treat Obstructive Hypertrophic Cardiomyopathy

Cytokinetics, Incorporated today announced that the U.S. Food & Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for Aficamten, a next-in-class cardiac myosin inhibitor, for the treatment of obstructive hypertrophic cardiomyopathy (oHCM). The FDA has assigned the NDA a standard review with a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2025. Notably, the FDA is not currently planning to hold an advisory committee meeting to discuss the application.

Publish Date: 02-12-2024 Source: Cytokinetics

Obstructive hypertrophic cardiomyopathy (oHCM), once known as idiopathic hypertrophic subaortic stenosis, is a condition commonly encountered. It stands as a significant contributor to sudden cardiac death among young individuals, including athletes, affecting both genders equally across diverse racial backgrounds. The familial form of oHCM follows an autosomal dominant inheritance pattern, making it the most prevalent genetically inherited cardiomyopathy. Additionally, it can manifest in individuals without a familial history due to de novo mutations. oHCM arises from gene mutations responsible for encoding sarcomere proteins, including B-myosin heavy chain, troponin, actin, and titin. These mutations induce structural irregularities in myofibrils and myocytes, potentially resulting in abnormal force generation and conduction issues. Consequently, in the absence of alternative causes, this leads to the characteristic phenotype of asymmetric left ventricular hypertrophy. Classification of oHCM can be based on whether it presents with obstruction or not. Diagnosis primarily relies on 2D echocardiography. Treatment strategies are tailored according to symptom severity. Lifestyle adjustments are advocated for mild cases, emphasizing the avoidance of vigorous physical exertion and heavy lifting. Initial pharmacological management for symptomatic oHCM typically involves beta blockers, although cardiac-selective calcium channel blockers may also be considered. It’s essential to steer clear of ACE inhibitors and nitrates in treatment regimens, as they can exacerbate left ventricular outflow tract obstruction by reducing afterload.

However, the current Obstructive Hypertrophic Cardiomyopathy (oHCM) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (Sotagliflozin, Mavacamten, IMB-1018972, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Obstructive Hypertrophic Cardiomyopathy (oHCM) treatment. The key companies in the advanced development stage are Lexicon Pharmaceuticals, Bristol-Myers Squibb, Imbria Pharmaceuticals, Inc., etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Obstructive Hypertrophic Cardiomyopathy (oHCM) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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