Dupilumab in addition to controller medications for children aged 6 to 11 years with asthma
Dupilumab is in clinical development as an add-on to controller medications for the maintenance treatment in children aged 6 to <12 years with uncontrolled, moderateto-severe asthma. Asthma is a common lung condition that causes wheezing, coughing and breathlessness. Individuals with asthma can suffer an asthma attack, which in severe cases can be fatal. Patients with …
Empagliflozin for Chronic Heart Failure with Preserved Ejection Fraction
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 …
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).
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.
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.
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.
Icosapent ethyl for reducing the risk of cardiovascular events
Icosapent ethyl is in clinical development as a treatment to reduce the risk of cardiovascular events in high-risk patients who have their cholesterol levels controlled with statin treatment, but have elevated triglycerides and other cardiovascular risk factors. Cardiovascular events include heart attack, angina and stroke. These diseases are the main cause of death in the UK, accounting for over a quarter of deaths each year. Patients receiving statin treatment are still at a high risk and would benefit from treatment to reduce cardiovascular events.
Levosimendan for respiratory function in amyotrophic lateral sclerosis
Levosimendan works through binding to a protein called troponin C, which sensitises cardiac and skeletal muscles to calcium and increases their force of contraction. This increased force of contraction is thought to increase diaphragm function and support respiratory dysfunction. Levosimendan is given as an oral capsule and if licensed, it will offer a treatment option for patients with ALS, potentially delaying the need for mechanical ventilation support.
Empaglifozin for treatment of chronic heart failure with reduced ejection fraction
Empagliflozin is given by mouth in the form of a tablet and works by inhibiting the sodium transporter NHE1. This prevents salt from being re-absorbed so there is increased excretion of salt from the body and a reduced volume of fluid in the blood vessels. It’s thought that these changes in sugar, salt and water metabolism in the body may contribute to the reductions in cardiovascular death. If licensed, empagliflozin may provide an additional treatment option for people with HFrEF who currently have limited therapies available.