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This search function provides links to outputs produced by NIHR Innovation Observatory. These are briefing notes or reports on new or repurposed technologies. This search will not return all technologies currently in development as these outputs are produced as required for our stakeholders.

Innovation Observatory > Reports > Cardiovascular Disease and Vascular Surgery

Results

Drugs

July 2020

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.

Drugs

July 2020

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.

Drugs

June 2020

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.

Drugs

June 2020

Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation in patients aged 12 years old and older

Elexacaftor and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The triple therapy of elexacaftor/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF heterozygous for F508del mutations, who currently have limited options.

Drugs

March 2020

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.

Drugs

November 2019

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.

Drugs

October 2019

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.

Drugs

August 2019

Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 6 to 11 years

The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is heterozygous for F508del mutation and a minimal function mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.

Drugs

August 2019

Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 6 to 11 years old

The triple fixed-dose combination (FDC), elexacaftor/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 6 to 11 years old. CF is the most common, life-limiting recessively inherited (a faulty gene inherited from both parents) disease in the UK. Genetic mutations affect the CF transmembrane conductance regulator (CFTR) gene, which is essential for the regulation of salt and water movements across cell membranes. These mutations mean that the CFTR protein is not processed and moved through the cells normally, resulting in little to no CFTR protein at the cell surface. This results in thickened secretions in organs with epithelial cell lining, mainly affecting the lungs and digestive system.

Drugs

August 2019

Ticagrelor in addition to acetylsalicylic acid for prevention of stroke in patients with acute ischaemic stroke or transient ischaemic attack

Ticagrelor in addition to acetylsalicylic acid (ASA) is in development for the prevention of new stroke in patients with acute ischaemic stroke or high-risk transient ischaemic attack. A stroke is a serious life-threatening medical condition that happens when the blood supply to part of the brain is cut off. There are three different types of stroke; ischaemic strokes, haemorrhagic strokes and transient ischaemic attacks. The aim of stroke therapy is to restore optimal blood flow to the brain, reduce any damage caused to the brain tissues, modulate any factors that may exacerbate this damage and if possible, repair the damage. Early treatment is critical to rescue potentially salvageable brain tissue.

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