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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

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.

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

Avapritinib for unresectable or metastatic gastrointestinal stromal tumours (GIST) who have been treated with at least 3 prior lines of therapy and GIST harbouring the PDGFRA D842V mutation regardless of prior therapy

Avapritinib is designed to potently and selectively inhibit the active forms of the KIT and PDGFRA mutant enzymes, and has shown potent activity in GIST driven by these gene mutations. If licensed, avapritinib will offer a fourth-line treatment option for patients with unresectable or metastatic GIST who have exhausted all other approved treatment options. It will also be the first licensed treatment option specifically for patients with unresectable or metastatic GIST harbouring a PDGRFA D842V mutation.

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

May 2019

Omalizumab for chronic rhinosinusitis with nasal polyps

Omalizumab is a monoclonal antibody that targets and blocks the immunoglobulin E (IgE). Human IgE is produced in large quantities in patients with allergies and triggers an allergic reaction in response to an allergen. By attaching to IgE, omalizumab ‘mops up’ the free IgE circulating in the blood. This means that when the body encounters an allergen, there is less IgE available to trigger an allergic reaction. This helps to reduce the symptoms of CRSwNP. If licensed, omalizumab will offer a treatment option as add-on therapy for patients with CRSwNP who have had an inadequate response to current standard-of-care treatments.

Drugs

May 2019

Setmelanotide for rare genetic obesity due to leptin receptor deficiency

Setmelanotide is in clinical development for the treatment of rare genetic obesity due to leptin receptor deficiency. The leptin receptor is found on the surface of cells in many organs and tissues of the body, including a part of the brain called the hypothalamus, which controls hunger and thirst. The leptin receptor is involved in …

Drugs

May 2019

Fenfluramine hydrochloride for treatment of seizures associated with Lennox-Gastaut syndrome

Fenfluramine belongs to a class of drugs called the selective serotonin releasing agonists which stimulates multiple 5-HT receptor sub-types through the release of serotonin. Fenfluramine may also act on other receptors and these actions may help to reduce the frequency of seizures. When added to other standard anti-epileptic treatments, fenfluramine hydrochloride has shown preliminary evidence of reducing seizure frequency. If licensed, fenfluramine hydrochloride may offer an additional treatment option for patients with Lennox-Gastaut syndrome.

Drugs

May 2019

Secukinumab for moderate to severe plaque psoriasis in children aged 6 to less than 18 years old

Secukinumab is a monoclonal antibody, a type of protein, designed to recognise and attach to a messenger molecule in the immune system called interleukin 17A. This molecule is involved in the inflammation and other immune system processes that cause psoriasis. By attaching to and blocking the action of interleukin 17A, secukinumab reduces the activity of the immune system and the symptoms of the disease. If licensed, secukinumab will offer a first-line systemic treatment option in children and adolescents with moderate to severe plaque psoriasis in patients who are candidates for systemic therapy.

Drugs

May 2019

Risdiplam for paediatric and adult patients with spinal muscular atrophy (SMA)

Risdiplam is an investigational, oral medicine that is designed to increase SMN protein levels in the central nervous system and throughout the body and thereby reducing the symptoms of the disease. If licensed risdiplam will offer a treatment option for paediatric and adult patients with SMA.

Drugs

May 2019

Inclisiran for primary hypercholesterolaemia and mixed dyslipidaemia

The current standard of care for patients with hypercholesterolaemia is primarily statins which are capable of reducing LDL‐C. There is however a subset of patients who are unable
to reach LDL‐C goals despite maximally tolerated oral lipid lowering therapy. Inclisiran is a medicinal product that inhibits a protein called PCSK9 especially present on the liver cells
leading to a decrease in circulating LDL‐C. If licensed, inclisiran will offer an additional treatment option for patients with primary hypercholesterolaemia as adjunctive therapy to diet and in combination with other lipid‐lowering therapies.

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