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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 > Drugs

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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

June 2019

Pembrolizumab in addition to platinum therapy and radiation for unresectable locally advanced head and neck squamous cell carcinoma

Pembrolizumab in addition to platinum therapy and radiation is in clinical development for the treatment of unresectable locally advanced head and neck squamous cell carcinoma. Cancers that are collectively known as head and neck cancers usually begin in the squamous cells that line the moist, mucosal surfaces inside the head and neck (for example, inside the mouth, the nose, and the throat). The main risk factors for squamous cell carcinomas are smoking tobacco and drinking alcohol. Symptoms may include sore throat, difficulty swallowing and pain in the ears and others. Treatment options for cancer that has spread usually involve chemotherapy such as (platinum-based therapy) and/or radiotherapy and focus on relieving symptoms and prolonging life rather than curing the cancer.

Drugs

June 2019

Benralizumab in addition to mometasone furoate for severe nasal polyposis

Benralizumab in addition to mometasone furoate is in development for the treatment of severe nasal polyposis/polyps. Nasal polyps are painless soft growths inside the nose. The exact cause is still unknown but asthma and a bad reaction to aspirin are known to increase the risk of developing the disease. Nasal polyps contain inflammatory fluid and …

Drugs

June 2019

Atezolizumab in combination with bevacizumab for untreated unresectable or advanced hepatocellular carcinoma

Atezolizumab in combination with bevacizumab is currently in clinical development for the treatment of patients with an unresectable or advanced type of liver cancer called hepatocellular carcinoma (HCC) that have not received previous treatment. HCC is the most common type of liver cancer and occurs mainly in patients with underlying chronic liver disease and cirrhosis. Advanced or metastatic HCC occurs when the cancer has spread to lymph nodes or to other organs. Advanced unresectable HCC is often diagnosed late in life and has a poor prognosis. It is a debilitating condition with many distressing symptoms, including pain, digestive problems and weight loss.

Drugs

June 2019

Spartalizumab in addition to dabrafenib and trametinib for unresectable or metastatic BRAF V600 mutant melanoma – first‐line

Spartalizumab in addition to dabrafenib and trametinib, is in clinical development for patients with previously untreated unresectable or metastatic BRAF V600 mutant
melanoma. Malignant melanoma is the most aggressive and life‐threatening form of skin cancer. General symptoms of advanced melanoma may include weight loss, loss
of appetite and fatigue. Factors associated with a higher risk of developing melanoma include a fair skin, exposure to sunlight and other sources of ultraviolet (UV) energy,
and a history of sunburn or moles.

Drugs

June 2019

Dupilumab for children aged 6 to less than 12 years with severe atopic dermatitis

Dupilumab is in development for the treatment of children aged ≥ 6 to < 12 years with severe atopic dermatitis (AD) uncontrolled with currently available therapies. AD is a chronic inflammatory skin disease that affects both children and adults and is characterised by redness, itchiness, and scaling of the skin. Some people only have small patches of dry skin, but others may experience widespread red, inflamed skin all over the body. Patients with moderate to severe AD could come across with sleep disturbances, anxiety, depression, and poor quality of life. Currently, the management of AD involves the removal or treatment of trigger factors that contribute to the development of the disease.

Drugs

June 2019

Ramucirumab in addition to erlotinib for EGFR mutation-positive metastatic non-small-cell lung cancer

Ramucirumab has been designed to attach to a receptor for a protein called Vascular Endothelial Growth Factor (VEGF). The VEGF receptor can be present at high levels in tumours and helps in the development of new blood vessels that supply the tumours. Ramucirumab blocks this action by blocking this receptor, thereby reducing the blood supply to the tumour and slowing the growth of the cancer. Erlotinib is currently a standard of care first-line treatment for locally advanced or metastatic NSCLC with EGFR mutations. It is thought that the addition of ramucirumab to erlotinib might improve its efficacy and provide an additional benefit by delaying/suppressing the EGFR mutation in patients with NSCLC.

Drugs

May 2019

Olaparib in combination with bevacizumab for ovarian, fallopian tube or primary peritoneal cancer – maintenance therapy

Olaparib belongs to a group of drugs called PARP enzyme inhibitors while bevacizumab is an anti-VEGF monoclonal antibody. Both drugs act in different but synergistic ways to kill tumour cells. It is thought that bevacizumab may increase the sensitivity of olaparib to killing the tumour cells. Olaparib administered orally as a monotherapy is already licensed as a maintenance therapy of advanced ovarian cancer. The addition of bevacizumab given by intravenous infusions may potentially improve treatment outcomes.

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