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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

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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.

Drugs

June 2019

Lumacafor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 to 23 months

The fixed-dose combination (FDC) lumacaftor/ivacaftor-FDC is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 12 to 23 months. 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

June 2019

VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older

The triple fixed-dose combination (FDC), VX-445/tezacaftor/ivacaftor-FDC, is in clinical development for cystic fibrosis (CF) that is homozygous for F508del mutation for patients aged 12 years and older. 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

June 2019

VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older

The triple fixed-dose combination (FDC), VX-445/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 12 years and older. 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

May 2019

Dapagliflozin for chronic heart failure with reduced ejection fraction

Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose co-transporter 2 (SGLT2). As blood is filtered by the kidneys, SGLT2 stops glucose in the bloodstream from being passed out into the urine. By blocking the action of SGLT2, dapagliflozin causes the kidney to pass out more glucose in the urine, thereby reducing the levels of glucose in the blood. Blood vessels can be damaged by the effects of high blood glucose levels and this can in turn cause damage to organs, such as the heart. Dapagliflozin may also increase the removal of fluid between tissue cells, contributing to reduced congestion with minimal impact on blood volume. If licensed, dapagliflozin may provide a treatment option for people with HFrEF who currently have limited therapies available.

Drugs

May 2019

Dapagliflozin for heart failure with preserved ejection fraction

Dapagliflozin blocks the action of a protein in the kidneys called sodium-glucose co-transporter 2 (SGLT2). As blood is filtered by the kidneys, SGLT2 stops glucose in the bloodstream from being passed out into the urine. By blocking the action of SGLT2, dapagliflozin causes the kidney to pass out more glucose in the urine, thereby reducing the levels of glucose in the blood. Blood vessels can be damaged by the effects of high blood glucose levels and this can in turn cause damage to organs, such as the heart. Dapagliflozin may also increase the removal of fluid between tissue cells, contributing to reduced congestion with minimal impact on blood volume. If licensed, dapagliflozin may provide a treatment option for people with HFpEF who currently have no effective therapies available.

Drugs

April 2019

Rivaroxaban for reducing the risk of major thrombotic vascular events in symptomatic peripheral arterial disease patients with a recent lower extremity revascularisation procedure

Rivaroxaban is an already approved oral medicine used for the prevention of thrombotic vascular events in different types of artery diseases including PAD. It acts by blocking specific pathways involved in the process of blood clots within blood vessels, reducing the risk of a major vascular event. Aspirin alone is the current standard treatment in patients with PAD undergoing revascularization procedures but its risk reduction may not be adequate. If licensed rivaroxaban will potentially be an option as an add-on therapy in this patient group.

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