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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

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Drugs

July 2019

Lacosamide for primary generalised tonic-clonic seizures – adjunctive therapy

Lacosamide is a medicinal product that is being developed for the treatment of Primary Generalised Tonic-Clonic Seizures (PGTCS). Epilepsy is a neurological disorder that is characterised by an imbalance in the excitation and inhibition of the brain and this imbalance causes a phenomenon known as a seizure. Seizures are brief increases in electrical activity within …

Drugs

July 2019

Niraparib for advanced ovarian, fallopian tube or primary peritoneal cancer – maintenance therapy

Niraparib as an oral formulation is in clinical development for maintenance therapy in patients with advanced ovarian, fallopian tube or primary peritoneal cancer following response to first-line platinum-based chemotherapy. Ovarian cancer includes a group of tumours that arise from diverse types of tissue contained in the ovary. The most common type of ovarian cancer arises …

Drugs

July 2019

Atezolizumab in addition to cobimetinib and vemurafenib for BRAF mutated metastatic melanoma

Atezolizumab in addition to cobimetinib and vemurafenib is in development for the treatment of BRAF mutated metastatic melanoma. Melanoma is a type of skin cancer which arises from the pigment cells (melanocytes) in the skin and is the most aggressive and life-threatening form of skin cancer. BRAF is a type of gene that drives rapid …

Drugs

July 2019

Nivolumab for oesophageal or gastro-oesophageal junction cancer – adjuvant

Nivolumab is a medicinal product that is being developed for the adjuvant treatment (after the primary treatment) of patients with resected oesophageal or gastro-oesophageal junction cancer. Oesophageal develops when abnormal cells in the gullet (oesophagus) grow in an uncontrolled way. The lower end of the oesophagus that joins the stomach is called the gastro oesophageal …

Drugs

July 2019

Imlifidase for kidney transplantation in highly sensitised patients with chronic kidney disease

Imlifidase is in clinical development for enabling kidney transplantation in highly sensitised patients with chronic kidney disease (CKD). CKD is a long-term irreversible condition where the kidneys do not work as well as they should. Kidney transplantation is considered to be treatment of choice for patients with end stage kidney disease. Many patients on the waiting list for organ transplantation carry antibodies to human leukocyte antigen (HLA), which is known as being ‘sensitised.’ Patients who are highly sensitised may find it difficult getting a donor and may not be able to receive a transplant due to increased risk of kidney rejection.

Drugs

July 2019

Atezolizumab for stage IV non-squamous or squamous non-small cell lung cancer

Atezolizumab is in clinical development for stage IV non-squamous or squamous non small cell lung cancer (NSCLC). NSCLC makes up the majority of lung cancers in the UK.
Stage IV (advanced/metastatic) NSCLC is when the cancer has spread beyond the lung which was initially affected, most often to the liver, the adrenal glands, the bones, and the
brain. Most patients with NSCLC are diagnosed at the advanced/metastatic stage where curative treatment with surgery is unsuitable. Currently, chemotherapy remains the main
first line option at this stage and often not well tolerated by many patients.

Drugs

July 2019

Ofatumumab for relapsing multiple sclerosis

Ofatumumab is in clinical development for the treatment of relapsing multiple sclerosis (MS). Multiple sclerosis is an autoimmune disease, meaning the body’s own immune cells (which usually fight infection) attack and damage the nerves and brain. This causes a range of issues including problems with walking, balance, memory and thinking as well as pain, tiredness and many other symptoms.

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.

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