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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

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.

Drugs

March 2019

Tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one residual mutation in patients aged 6 to 11 years

Tezacaftor is 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 combination therapy of tezacaftor/ivacaftor-FDC (Symkevi®) has been approved in the EU for patients aged 12 years and older with CF that have one of these gene mutations. If approved, this licence extension would mean that patients with these gene mutations could have access to this treatment regimen at an earlier age.

Drugs

March 2019

VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older

VX-659 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 VX-659/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF with F508del mutations, who currently have limited options.

Drugs

March 2019

Tezacaftor and ivacaftor for cystic fibrosis homozygous or heterozygous for F508del mutation in patients aged 12 years and older who discontinued treatment with Orkambi

Tezacaftor is 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 combination of tezacaftor and ivacaftor may result in an effective treatment for people with CF and may especially be a treatment option for those who cannot take a combination of ivacaftor and lumacaftor (another cystic fibrosis medicine), due to side effects or interactions with other medicines they are taking.

Drugs

March 2019

VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older

VX-659 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 VX-659/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF with F508del mutations, who currently have limited options.

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