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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 > Drugs

Results

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

Brigatinib for ALK-positive, locally advanced or metastatic, non-small cell lung cancer previously treated with alectinib or ceritinib

Brigatinib is a medicinal product that is being developed for the treatment of patients with locally advanced or metastatic ALK-positive non-small cell lung cancer (NSCLC) whose disease have progressed following treatment with alectinib or ceritinib. NSCLC is the most common type of lung cancer although a small proportion of NSCLC patients have a rearrangement in the ALK gene. Locally advanced or metastatic cancer means cancer has spread outside the lungs where it started, to other parts of the body and cannot be cured. Current treatment with drugs such as alectinib or ceritinib are effective in slowing the disease and helping patients to live longer, although some patients eventually develop treatment resistance and will require other therapies.

Drugs

August 2019

Belantamab mafodotin for relapsed / refractory multiple myeloma – Fourth line

Belantamab mafodotin is in clinical development for the treatment of multiple myeloma (MM) in patients who are refractory or have relapsed to prior treatments. MM is a rare, incurable cancer of the plasma cells in the bone marrow where large amounts of abnormal plasma cells are produced and interfere with the production of red and white blood cells and platelets. People with MM will experience periods of time without symptoms followed by periods when the illness comes back (‘relapsed’ MM). Eventually the periods without symptoms will shorten and the illness will become immune to the drugs given to treat it (‘refractory’ MM).

Drugs

August 2019

Romiplostim for idiopathic thrombocytopenic purpura in adult patients who are refractory to other treatments

Romiplostim is a medicinal product that is being developed for the treatment of adult patients with idiopathic thrombocytopenic purpura (ITP) who are refractory to other treatments. ITP is the condition of having a low platelet count due to unknown cause. It is also known as immune thrombocytopenic purpura. Many people with ITP do not have symptoms, however people with very low platelet count can have symptoms such as pin prick rash, easy bruising, nosebleeds, gum bleeds, black mouth blisters, fatigue, and heavy periods. Most of the currently available treatments have significant side effects with some treatments leaving patients are at increased risk of infections.

Drugs

August 2019

Nivolumab and relatlimab for untreated advanced or metastatic melanoma – first line

Nivolumab works by improving the activity of white blood cells increasing the ability of the immune system to kill cancer cells. Relatlimab has the potential to increase the immune system response and kill cancer cells. If licenced for this indication, a fixed dose combination of intravenous nivolumab and relatlimab may provide a new therapeutic option for untreated patients that shows similar adverse effects than treatment with nivolumab alone.

Drugs

August 2019

Trastuzumab deruxtecan for HER2-positive metastatic or unresectable breast cancer

Trastuzumab deruxtecan is in clinical development for the treatment of adults with HER2-positive, unresectable and/or metastatic breast cancer who have previously received anti-HER2 therapies. HER2-positive breast cancer is when the cancer tests positive for HER2 protein, which promotes the growth of cancer cells and tend to be more aggressive than other types of breast cancer. Metastatic breast cancer (stage IV) is when the cancer has spread beyond the breast and nearby lymph nodes to other organs in the body while unresectable means that the cancer cannot be treated by surgery. Treatment of the disease often involve the use of anti-HER2 therapies, chemotherapy or a combination of both.

Drugs

August 2019

Pexidartinib for tenosynovial giant cell tumour

Pexidartinib is a medicinal product that is being developed for the treatment of adult patients with symptomatic tenosynovial giant cell tumour (TGCT). TGCT is also referred to as giant cell tumour of the tendon sheath (GCT-TS) or pigmented villonodular synovitis (PVNS). TGCT is a rare, neoplasm derived from the thin layer of tissue that lines the joints and tendons leading to the formation of a mass. TGCT normally affects young adults of both sexes and is associated with severe morbidity and functional limitation. Surgery is currently the standard treatment although some cases of TGCT have a poorer likelihood of successful cure with surgery due to the high risk of recurrence. There are no current systemic treatment for symptomatic TGCT.

Drugs

August 2019

Axicabtagene ciloleucel for relapsed/refractory diffuse large B-cell lymphoma – second-line

Axicabtagene ciloleucel is in clinical development as second-line treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL). DLBCL is a cancer affecting a type of white blood cells called lymphocytes or B-cells. DLBCL is an aggressive cancer and although it can be cured in more than half of people affected, it remains a serious and life threatening disease. A relapse is when the lymphoma comes back after successful treatment and refractory means the lymphoma did not respond to the first course of treatment.

Drugs

August 2019

Pembrolizumab in addition to chemotherapy for perioperative treatment of gastric or gastro-oesophageal junction adenocarcinoma

Pembrolizumab in addition to chemotherapy is in clinical development for gastric or gastro-oesophageal junction cancer. Gastric cancer is cancer that starts anywhere inside the stomach or the stomach wall. Initial symptoms of disease are similar to other stomach conditions but symptoms of advanced stages may include a lack of appetite and subsequent weight loss; fluid in the abdomen and blood in the stool. Because of the nature of symptoms, gastric cancer is often diagnosed at an advanced stage. Surgery is a treatment option and often combined with chemotherapy given before (neo-adjuvant) and after (adjuvant) the surgery to improve treatment outcomes.

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