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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

Results

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.

Drugs

February 2020

Finerenone for chronic kidney disease in patients with type 2 diabetes mellitus

Finerenone is selective inhibitor of a specific protein, which blocks the damaging effects of hormones (such as aldosterone and cortisol) which can cause damage to the heart and kidneys in diabetic patients. Finerenone is administered orally in tablet form, and evidence suggests it may offer organ protection compared to similar inhibitors which are currently used. If licensed, finerenone will offer patients with type 2 diabetes mellitus who have chronic kidney disease an add-on therapy to slow down long-term kidney damage and reduce the adverse impact on the structure and function of the heart and vessels.

Drugs

February 2020

ATB200/AT2221 for Late onset Pompe disease in adults and adolescents >12 years – first line

ATB200 (cipaglucosidase alfa) is a recombinant human acid alfa glucosidase (rhGAA) enzyme that shows significantly improved uptake into muscle cells compared with algucosidase alfa at similar doses and translates into optimised glycogen reduction in disease-relevant muscles in non-clinical models. AT2221 (miglustat) is the pharmacological chaperone that stabilises and enhances the plasma exposure of ATB200, resulting in improved delivery of active enzyme to key disease-relevant tissues. If licensed, ATB200/AT2221 will offer an additional treatment option for patients with Pompe disease with potentially improved efficacy.

Drugs

January 2020

Daratumumab in addition to cyclophosphamide, bortezomib and dexamethasone for newly diagnosed systemic amyloid light-chain amyloidosis

Daratumumab in addition to cyclophosphamide, bortezomib and dexamethasone (CyBorD) is in clinical development for newly diagnosed systemic amyloid light-chain (AL) amyloidosis in adults. AL amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in tissues and organs such as the kidneys, liver, gut, heart and nerves. In AL amyloidosis, the deposits are made up of proteins (called immunoglobulin light chains) produced in excess by malfunctioning white blood cells in the bone marrow.

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.

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