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.
Gefapixant for chronic refractory cough – First line
Gefapixant is an oral drug that blocks receptors in the sensory cells in the airways and lungs, potentially reducing cough frequency in patients with refractory or unexplained chronic cough. Preliminary results from early studies have demonstrated that gefapixant is efficacious and safe. If licenced, gefapixant could provide a treatment option for patients with refractory or unexplained cough who have no licensed therapies available and few effective therapies available.
Vericiguat for chronic heart failure with reduced ejection fraction
Vericiguat is a medicinal product in clinical development for the treatment of heart failure (HF) with reduced ejection fraction. HF is a complex clinical syndrome of symptoms and signs that suggest the efficiency of the heart as a pump is impaired. Symptoms include breathlessness and fatigue, and signs of the condition include swollen ankles and crackling sounds in the lungs. More than half of people with HF have a reduced ejection fraction (HFrEF), also referred to as systolic heart failure, where the heart muscle does not contract effectively, and therefore less oxygen-rich blood is pumped out to the body. There remains a large unmet need for new therapies in the treatment of HFrEF.
Fevipiprant maintenance therapy for uncontrolled asthma – add on therapy
Fevipiprant is in clinical development for the treatment of patients aged 12 years and older with uncontrolled asthma who remain symptomatic despite treatment with inhaled corticosteroids (ICS) with or without at least one additional controller. Whilst there is no cure for asthma, most patients are able to control their symptoms by taking daily preventative medication and additional controllers when required. However, a small subset of asthma patients are resistant to the current standard of care for asthma and are unable to control their symptoms. This can have severe implications on their quality of life as uncontrolled asthma can result in decreased physical fitness, decreased sleep quality and decreased productivity at work or school.