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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 > Endocrine, Nutritional and Metabolic

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Drugs

November 2019

Treosulfan in combination with fludarabine for paediatric non-malignant disease before allogeneic stem cell transplant

Treosulfan in addition to fludarabine is in clinical development for paediatric non-malignant disease prior to allogeneic stem cell transplant. Treosulfan is a medicine given to patients before they have a bone marrow transplant from a donor known as ‘allogeneic haematopoietic stem cell transplantation’. It is used as a ‘conditioning’ treatment to clear the patient’s bone marrow and make room for the transplanted bone marrow cells, which can then produce healthy blood cells. Treosulfan is used together with another medicine called fludarabine for the treatment of a variety of disorders that require a bone marrow transplant.

Drugs

November 2019

Avalglucosidase alfa for late-onset Pompe disease

Avalglucosidase alfa is in clinical development for the treatment of late-onset Pompe disease. Pompe disease is an inherited, genetic disorder which results in the deficiency of the enzyme ‘acid alpha-glucosidase’ (GAA). This deficiency leads to progressive accumulation of glycogen, a type of sugar, usually stored in muscle tissues particularly around the heart, skeletal muscle and respiratory muscles. Late-onset Pompe disease develop after one year of age, and is a serious, progressive, debilitating, and ultimately life threatening disease associated with high morbidity. Enzyme replacement therapy with alglucosidase alfa is a recommended treatment approach but the main drawback of the current option is the limited uptake by affected muscles leading to limited clinical benefits in some patients.

Drugs

October 2019

Migalastat for Fabry disease in children aged 12 to 15 years

Migalastat works by stabilizing the body’s own dysfunctional enzyme, so it can clear the accumulated disease substrate in patients who have amenable mutations. Migalastat is currently licenced for patients aged 16 or over with Fabry disease and an amenable mutation. If the license is extended, migalastat may offer an additional treatment option for paediatric subjects 12 to 15 years old with Fabry disease and an amenable mutation, who weight over 45Kgs.

Drugs

August 2019

Nitisinone for Alkaptonuria

Nitisinone is in clinical development for the treatment of alkaptonuria. Alkaptonuria is a rare metabolic disorder, in which patients lack a functional enzyme that prevents the body fully breaking down two amino acids called tyrosine and phenylalanine. This results in a build-up of a chemical called homogentisic acid (HGA) in the body, which is deposited as black pigment in tissues, in a process called ochronosis. This results in dark colouration of urine, joint problems, breathing difficulties and heart, kidney and prostate problems. In the later stage, patients may experience physical disability and inability to perform daily activities. Early recognition and management of alkaptonuria is desirable to slow the progression of this disease.

Drugs

August 2019

Evinacumab for homozygous familial hypercholesterolemia

Evinacumab is in clinical development for the treatment of homozygous familial hypercholesterolemia (HoFH). Familial hypercholestrolaemia (FH) is an inherited condition where a person’s cholesterol levels are higher than normal from birth as the liver is unable to break down or remove excess cholesterol. Specifically, FH patients have severe elevations in low density lipoprotein cholesterol (LDL-C) …

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.

Drugs

May 2019

Canagliflozin for chronic kidney disease in adult patients with type 2 diabetes mellitus

Canagliflozin belongs to a class of antidiabetic drugs called SGLT2 inhibitors that act by encouraging the body to filter out more glucose from the blood and excrete it via the urine. Canagliflozin is already licensed for use in type 2 diabetes mellitus but emerging evidence has also suggested that it offers substantial kidney protection, by slowing the progression of diabetic kidney disease. Canagliflozin will offer patients with type 2 diabetes mellitus who have chronic kidney disease a single treatment option to improve both kidney function alongside blood glucose control.

Drugs

April 2019

Liraglutide for weight management in obese adolescents aged 12-17 years old

Liraglutide acts like a hormone the body produces naturally that regulates appetite, known as glucagon-like-peptide (GLP-1). By activating areas of the brain that regulate appetite, liraglutide may decrease feelings of hunger which can lead to lower calorie intake and weight loss. If licensed, liraglutide may improve long-term outcomes for weight management in obese adolescents 12-17 years old who currently have limited treatment options.

Drugs

April 2019

Sotagliflozin for type 2 diabetes mellitus

Sotagliflozin is one of a new class of drugs that increase elimination of glucose in the urine. They offer the advantages of a reduced risk of low blood sugar. Sotagliflozin reduces the reabsorption of glucose from the intestines and kidneys, thus improving blood glucose control in diabetes mellitus. If licensed, sotagliflozin will offer an additional treatment option for patients with T2DM.

Drugs

February 2019

LY-900014 (ultra-rapid lispro) for adults with type 1 diabetes mellitus

LY-900014 (ultra-rapid lispro) is in development for the treatment of adult patients with type 1 diabetes mellitus. Type 1 diabetes mellitus is a condition which usually starts early in life and occurs as a result of the pancreas not producing enough (or sometimes any) insulin. This results in elevated blood sugar levels which can damage many organs in the body. Type 1 diabetes usually runs in families. While type 1 diabetes cannot be cured, having regular, at least daily, injections of insulin can keep blood sugar levels stable. However some people, despite having regular injections of insulin, still do not have stable blood sugar levels.

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