January 2021
Mavacamten for hypertrophic cardiomyopathy
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).
December 2020
Treprostinil for chronic thromboembolic pulmonary hypertension
Treprostinil is in clinical development for patients with chronic thromboembolic pulmonary hypertension (CTEPH). CTEPH is a progressive disease caused by blood clots forming in the lungs that do not dissolve. These clots restrict blood flow through the lungs and cause scar tissue to form on the inside of the pulmonary arteries (arteries that supply the lungs) resulting in pulmonary hypertension. This means the heart has to work harder to pump the blood and can weaken the heart muscles. Some patients may be offered surgery to remove the scar tissue but in patients who are inoperable or who have persistent/recurrent CTEPH after surgery there is a need for additional treatment options.
November 2020
Tezepelumab for severe, uncontrolled asthma
Tezepelumab is a monoclonal antibody (protein) that targets proteins early in the inflammatory cascade. By targeting proteins early in the inflammatory cascade, several downstream inflammatory proteins are suppressed, thereby decreasing inflammation. Tezepelumab given as an add-on-therapy to patients with severe uncontrolled asthma has been shown to be safe, well tolerated, and effective. Tezepelumab is administered subcutaneously. If licensed, tezepelumab as an add on therapy would offer an additional biological therapy to those over 12 years old, with severe asthma.
November 2020
Omecamtiv mecarbil for treating chronic heart failure with reduced ejection fraction
Omecamtiv mecarbil is given by oral administration in the form of a tablet. Omecamtiv mecarbil works by interacting with a protein called cardiac myosin that is responsible for converting chemical energy into the mechanical force that results in contraction of the heart. The interaction of omecamtiv mecarbil with cardiac myosin improves the performance of the heart muscle to preserve its function. If licensed, omecamtiv mecarbil may provide an additional treatment option for people with HFrEF who currently have limited therapies available.