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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

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Drugs

September 2019

Carfilzomib in addition to daratumumab and dexamethasone for relapsed and/or refractory multiple myeloma

Carfilzomib, is a proteasome inhibitor. Proteasome is a system within the cells that breaks down proteins that are no longer needed. Cancer cells have an increased need to produce and break down proteins as they multiply rapidly. When carfilzomib stops the proteasome from breaking down proteins in the cancer cells, the proteins build up and cause the cells to die, slowing down the growth of the cancer. The addition of daratumumab and dexamethansone to carfilzomib may potentially improve outcomes and reduce side effects in patients with relapsed and/or refractory MM who have received prior therapies.

Drugs

September 2019

Atezolizumab in addition to paclitaxel for inoperable, locally advanced or metastatic triple negative breast cancer – first-line

Atezolizumab as an intravenous infusion in combination with an intravenous infusion of paclitaxel (chemotherapy) is in clinical development for the first-line treatment of locally advanced or metastatic triple-negative breast cancer (TNBC). TNBC is a type of breast cancer in which the cancer cells do not express receptors for oestrogen or progesterone or HER2 protein. Treatment of TNBC is challenging because of a lack of targeted therapy, aggressive disease course, and relatively poor prognosis. Treatment is usually through a combination of surgery, radiotherapy, and chemotherapy.

Drugs

September 2019

Ixazomib citrate for newly diagnosed multiple myeloma inelgible for autologous stem cell transplant– maintenance therapy

Ixazomib citrate is a novel oral medicinal product that is already licensed in the UK for the treatment of MM in patients who have received at least one prior therapy (in combination with lenalidomide and dexamethasone). Ixazomib citrate offers the potential advantage over similar medicines in its class of being more effective in its anticancer activity, reduced side effects and more convenient to administer (through its weekly oral dosing). If approved as maintenance therapy for NDMM patients, ixazomib maintenance has the potential to prolong the time patients live without their disease getting worse (progression free survival) as well as offering more convenient oral dosing that allows long term administration and improvement of patients’ quality of life.

Drugs

September 2019

NOVOCART 3D for articular cartilage defects of the knee

NOVOCART 3D Autologous Chondrocyte Transplantation System is a biologic-device combination product composed of autologous (the patient’s own) chondrocytes (cartilage producing and maintaining cells) seeded on a bioresorbable three-dimensional collagen scaffold, which is secured directly into the defect. The use of NOVOCART 3D involves two separate surgeries. In the first surgery, a small amount of knee cartilage is removed from the patient’s affected knee and sent to the company to be processed for re-implantation. In a second surgery, the damaged area of the knee cartilage is cleaned and the sponge-like scaffold containing the autologous chondrocytes is implanted. If licensed, NOVOCART 3D will offer an additional treatment option for repair of articular cartilage defects of the knee.

Drugs

September 2019

Ibrutinib in addition to rituximab for chronic lymphocytic leukaemia – first-line

Ibrutinib works against cancerous B lymphocytes, which are a type of white blood cells affected by these diseases. It does this by blocking an enzyme called Bruton’s tyrosine kinase (BTK), which promotes survival of B lymphocytes and their migration to the organs where these cells normally divide. By blocking BTK, ibrutinib decreases the survival and migration of B lymphocytes, thereby delaying the progression of cancer. Ibrutinib is available in tablets taken orally. If licensed, ibrutinib in addition to rituximab will offer an additional first-line treatment option for untreated young and fit patients with CLL or SLL.

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.

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