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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

February 2021

Tirzepatide for treating type 2 diabetes mellitus

Tirzepatide is a new type of drug that is administered by subcutaneous injection and acts on two proteins known as the gastric inhibitory peptide (GIP) and glucagon-like peptide-1 (GLP-1) and results in more insulin being released from the pancreas. Tirzepatide has the potential to improve blood sugar levels and increase weight loss compared to current treatment options. If licenced, tirzepatide will offer an additional treatment option for patients with T2DM.

Drugs

February 2021

Setmelanotide for obesity in Bardet-Biedl syndrome and Alström syndrome

Setmelanotide is a protein that binds to a specific receptor to activate areas in the brain that control appetite. This reduces hunger sensations and therefore obesity. This is important in BBS and AS as obesity and hyperphagia are symptoms in both syndromes and these conditions can have damaging effects on the health of patients. If licensed, setmelanotide, given as an injection under the skin, will offer a first pharmacological treatment for BBS and AS.

Drugs

November 2020

Leriglitazone for X-linked andrenoleukodystrophy

Leriglitazone is expected to work in patients with ALD by activating receptors called ‘PPAR gamma’, found in the mitochondria, which are components within cells that generate energy. Leriglitazone will improve the ALD pathogenic cascade by improving mitochondrial dysfunction, reducing oxidative stress and neuroinflammation, promoting demyelination and halting axonal degeneration. Hence, leriglitazone is expected to protect cells from damage and slow the progression of the disease. If licensed, leriglitazone would offer the first drug treatment option for adult ALD patients with AMN who currently have no effective therapies available.

Drugs

November 2020

Pegunigalsidase alfa for Fabry disease – first-line

Pegunigalsidase alfa is produced by a method known as ‘recombinant DNA technology’: it is made by cells into which a gene (DNA) has been introduced, which makes them able to produce the enzyme. The replacement enzyme has also been modified to reduce the rate at which it is removed from the body, allowing it to act for longer. Pegunigalsidase alfa was designed to increase amount of medicine in the blood and reduce the ability of a substance to provoke an immune response, thereby enhancing efficacy compared with available products. If licensed, pegunigalsidase alfa would offer an additional first-line, long -term ERT options for adult and paediatric patients (>3 yr) diagnosed with FD.

Drugs

August 2020

Semaglutide for overweight individuals

Semaglutide is in clinical development for the treatment of overweight and obese individuals. Excess weight can place stress on both mental and physical health, leading a number of complications such as depression, low self-esteem, and increased risk of heart disease, stroke and type 2 diabetes. Weight can be affected by a number of factors such as diet, physical activity, genetics and general health conditions, however diet and exercise are the two main contributing factors. Being overweight is reversible through lifestyle changes such as increased exercise, healthy diet and a net calorie deficit, along with help through counselling and medication. If weight is not able to be controlled, surgery may be required. Therefore, there is an unmet need for pharmacological therapies that target both weight and glucose control.

Drugs

August 2020

Triheptanoin for long chain fatty acid oxidation disorders

Triheptanoin (UX007) is being developed for the treatment of long chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is a group of six rare genetic disorders in which the body is unable to convert dietary fatty acids into energy. This inability to produce energy from fat can lead to severe depletion of glucose in the body and serious, unpredictable complications, which can lead to hospitalizations or early death despite the best current care. LC-FAOD has no specifically approved treatments. The current disease management includes avoidance of fasting, maintenance of a low fat diet, and supplementation of diet with oils rich in essential fatty acids.

Drugs

July 2020

Ozanimod for moderate to severe ulcerative colitis

Ozanimod, administered orally, is a new sphingosine 1-phosphate (S1P) receptor modulator. Treatment with S1P modulators is believed to work by interfering with signalling pathways that contribute to tissue inflammation. If licensed, ozanimod will offer an additional treatment option for adult patients with moderate to severe UC who have had an inadequate response, lost response to, or were intolerant to conventional therapy or a tumour necrosis factor-alpha (TNF) antagonist. In one phase II clinical trial, ozanimod at a daily dose of 1mg resulted in a slightly higher rate of clinical remission of ulcerative colitis than placebo.

Drugs

May 2020

Veverimer for metabolic acidosis associated with chronic kidney disease

Veverimer is given as an oral suspension and it works by binding to hydrochloric acid in the gastrointestinal tract and removing it from the body through excretion in the faeces. Preliminary results from early studies have demonstrated that veverimer is efficacious and safe. If licensed, veverimer could offer an additional treatment option for patients with metabolic acidosis associated with CKD.

Drugs

March 2020

Eladocagene exuparvovec for aromatic l-amino acid decarboxylase deficiency

Eladocagene exuparvovec is a type of gene replacement therapy which involves the transfer of the gene encoding the production of the enzyme needed by the brain for the formation of dopamine and serotonin. The gene therapy is injected via a surgical procedure into an area of the brain called the putamen. By increasing production of the AADC-enzyme, this therapy increases dopamine production in the target area of the brain and improves motor and cognitive symptoms in patients. If licensed, eladocagene exuparvovec will provide the first medicinal treatment option for adult and child patients with AADC-deficiency, a disease of very high unmet clinical need.

Drugs

February 2020

Tralesinidase alfa for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B)

Tralesinidase alfa is an investigational enzyme replacement therapy (ERT) designed to replace the faulty enzyme with a healthy one in patients with MPS IIIB. Tralesinidase alfa is delivered directly to the fluid surrounding the brain (cerebrospinal fluid). If licensed, tralesinidase alfa will offer new therapy option for patients with MPS IIIB who currently have no treatment options.

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