logo
Menu

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

June 2020

Empaglifozin for treatment of chronic heart failure with reduced ejection fraction

Empagliflozin is given by mouth in the form of a tablet and works by inhibiting the sodium transporter NHE1. This prevents salt from being re-absorbed so there is increased excretion of salt from the body and a reduced volume of fluid in the blood vessels. It’s thought that these changes in sugar, salt and water metabolism in the body may contribute to the reductions in cardiovascular death. If licensed, empagliflozin may provide an additional treatment option for people with HFrEF who currently have limited therapies available.

Drugs

June 2020

Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation in patients aged 12 years old and older

Elexacaftor and tezacaftor are designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein. Ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface. The triple therapy of elexacaftor/tezacaftor/ivacaftor-FDC may result in an effective therapeutic option for people with CF heterozygous for F508del mutations, who currently have limited options.

Drugs

June 2020

Abilify MyCite (aripiprazole with sensor) for bipolar disorder and schizophrenia

Abilify MyCite is a drug-device combination comprising aripiprazole embedded with an Ingestible Event Marker (IEM) sensor. The aripiprazole attaches in the brain to receptors which help normalise the activity of the brain. The drug is packaged within an innovative system which helps patients (and healthcare workers) monitor when the medication is taken through integration with a sensor patch and app. Abilify MyCite is different to other ways of monitoring whether medication has been taken because it gives results of the actual medication taken, without need for blood or urine samples, as opposed to an estimate and if licensed, will offer an additional treatment option for patients with BP1 and SCH.

Drugs

June 2020

Potassium citrate and potassium bicarbonate for distal renal tubular acidosis – first line

ADV7103 is an innovative product with a prolonged-release formulation, designed to maintain sustained release over a twelve-hour period for dRTA treatment. As the combination is alkaline (pH greater than 7) and contains potassium, it is expected to neutralise excess acid in the blood and restore levels of potassium. The product was developed as a multi particulate formulation in 2mm granules that contains two active pharmaceutical ingredients. Bicarbonate + potassium citrate is tasteless and easy to administer, in small-size format that offer flexible, personalized dosing which makes it easier to take for patients of all ages. The ability of ADV7103 to normalize biological disorders caused by dRTA throughout the course of treatment has been shown in a Phase III extension study. If licensed, ADV7103 will offer a potentially curative treatment option for patients with dRTA, who currently have few approved therapies available.

Drugs

June 2020

Nivolumab in combination with ipilimumab for previously untreated unresectable or metastatic urothelial cancer – first-line

Nivolumab in combination with ipilimumab is in clinical development for PD-L1 positive patients with previously untreated unresectable or metastatic urothelial cancer regardless of cisplatin eligibility. Urothelial cancer, a subset of bladder cancer, occurs on the lining of the renal pelvis, ureter, bladder and urethra, and other parts of the urinary system. In some cases,the tumour spreads into the surrounding muscles or other parts of the body which means that it cannot be cured by surgery. Metastatic urothelial cancer occurs when the cancer has spread to other parts of the body, such as the liver or bones. The symptom of urothelial cancer is blood in the urine, but symptoms may only appear once the cancer grows larger or into the deeper layers of the bladder wall for both men and women. Other symptoms may include increased frequency/urgency/pain of urine passing, weight loss, back/lower tummy/bone pain, fatigue and illness.

Drugs

June 2020

Tucatinib in addition to trastuzumab and capecitabine for advanced breast cancer

Tucatinib is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. By inhibiting HER2, it is hoped tucatinib will inhibit the growth of HER2-positive tumours. If licenced, tucatinib in addition to trastuzumab and capecitabine will provide an additional treatment option for adults with advanced HER2 positive metastatic breast cancer.

Drugs

June 2020

Dusquetide for chemoradiation-induced oral mucositis in patients with squamous cell carcinoma of the head and neck

Dusquetide is in clinical development for the treatment of chemoradiation induced oral mucositis (OM) in patients with squamous cell carcinoma of the head and neck. OM is the most common toxicity in patients with head and neck cancer who are treated with chemoradiation, which not only impairs their function and quality of life but also …

Drugs

June 2020

Etrolizumab for treating moderately to severely active ulcerative colitis in adults

Etrolizumab is a new monoclonal antibody (an immune protein) delivered by subcutaneous injection. The treatment works by targeting molecules called integrins to control the immune response and prevent the accumulation of immune molecules, which cause inflammation in individuals with a form of ulcerative colitis where inflammation is not mediated by a signalling protein called tumour necrosis factors (TNF) alpha (‘non-TNF-α’) and who are therefore intolerant to TNF blockers. This represents a new target group as current therapies focus mainly on anti-TNF inflammation. In one study, etrolizumab showed a greater reduction of intestinal lymphocyte infiltration in comparison to standard treatment.

Drugs

May 2020

JNJ-68284528 for relapsed or refractory multiple myeloma

JNJ-68284528 is a type of gene therapy that targets a protein called BCMA (B cell maturation antigen) which is expressed on B-cells (a type of immune cell). Binding of JNJ-68284528 to BCMA prevents B-cell maturation and differentiation into plasma cells. JNJ-68284528 is administered as an intravenous infusion. Early stage clinical trials (phase II) have demonstrated both safety and efficacy of JNJ-68284528 in patients with relapsed/refractory MM and could therefore provide an additional treatment option for patients with the disease.

Drugs

May 2020

Pembrolizumab in addition to lenvatinib for PD-L1 positive (≥1%) non-small-cell lung cancer – first line

Lenvatinib is a tyrosine kinase inhibitor that targets several different growth factor receptors including vascular endothelial growth factor (VEGFR) and fibroblast growth factor receptors (FGFR). By blocking these receptors, lenvatinib can reduce tumour growth. Pembrolizumab is a drug that works by improving the activity of white blood cells in killing cancer cells by blocking a protein, PD-L1. If licenced, lenvatinib (administered orally) in addition to pembrolizumab (administered intravenously) could provide an additional treatment option for adults with PD-L1 positive NSCLC.

Get Alerts