Empagliflozin is in clinical development for chronic heart failure and preserved ejection fraction (HFpEF). Heart failure (HF) is a clinical syndrome caused by the impaired ability of the heart to cope with the metabolic needs of the body. This results in breathlessness, fatigue, and fluid retention. The European Society of Cardiology(ESC) defines HFpEF as the presence of signs and symptoms of HF, LVEF ≥50%, elevated natriuretic peptides (NP) levels, and structural heart disease and/or diastolic dysfunction. HFpEF is a growing problem affecting more than half of the patients with HF. HFpEF has a significant morbidity and mortality rate and so far, no treatment has been demonstrated to improve the outcomes in HFpEF.
Empagliflozin is administered orally and usually taken once daily in the morning. Empagliflozin inhibits a protein found in the kidney called sodium-glucose co-transporter-2 (SGLT-2). It blocks glucose absorption in the kidney and increases the amount of glucose excreted in the urine. Empagliflozin significantly reduces the risk of cardiovascular events and heart failure hospitalisation. If licensed, empagliflozin may offer an additional treatment option for patients with HFpEF who currently have no effective treatments available.
Mavacamten is currently being developed for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults. Hypertrophic cardiomyopathy (HCM) is a genetic condition whereby areas of heart muscle become thickened and stiff. Blood decreases in the left ventricular volume and narrowing of the left ventricular outflow tract (LVOT) is classified as obstructive HCM (oHCM). HCM is a genetic condition that is caused by a change or fault (or mutation) in one or more genes. The most common symptoms are shortness of breath, palpitations, chest pain and light-headedness. Patients with oHCM can develop serious complications such as atrial fibrillation, heart failure, malignant ventricular arrhythmias, and sudden cardiac death (SCD).