Search All Tech Briefings

Search technology briefings produced by NIHR Innovation Observatory. These are short reports on new or repurposed medicines that we have already sent to our stakeholders. Only those technologies (medicines) that are within the scope of our stakeholders will have a technology briefing available. Search our technology briefings by intervention (drug or device), specific indication (disease or condition), or general therapeutic area.

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Title	Intervention	Indication	Therapeutic Area	Year	Actions
Mitapivat for treating pyruvate kinase deficiency	Mitapivat (AG-348)	Pyruvate kinase deficiency	Endocrine Nutritional and Metabolic Disorders	2020	View \| Download
NEOD001 for amyloid light-chain (AL) amyloidosis	Birtamimab (NEOD001)	Amyloidosis	Endocrine Nutritional and Metabolic Disorders	2017	View \| Download
Nitisinone for Alkaptonuria	Nitisinone (Orfadin; SYN118)	Alkaptonuria	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2019	View \| Download
Olezarsen for treating familial chylomicronaemia syndrome	Olezarsen (ISIS 678354; AKCEA-APOCIII-LRx)	Familial chylomicronaemia syndrome (FCS)	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2024	View \| Download
Oral Semaglutide for the treatment of type 2 diabetes mellitus	Semaglutide (Ozempic)	Type 2 diabetes	Endocrine Nutritional and Metabolic Disorders	2018	View \| Download
OTL-200 for Metachromatic Leukodystrophy	OTL-200 (GSK-2696274)	Metachromatic leukodystrophy (MLD)	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2019	View \| Download
Pariglasgene brecaparvovec for treating glycogen storage disease type 1a	Pariglasgene brecaparvovec	Glycogen storage disease type 1a	Endocrine Nutritional and Metabolic Disorders	2023	View \| Download
Pegunigalsidase alfa for Fabry disease – first-line	PRX-102 (Pegunigalsidase alfa)	Fabry disease	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2020	View \| Download
Pegzilarginase for Arginase-1 deficiency	Pegzilarginase (AEB1102; Loargys)	Hyperargininemia	Endocrine Nutritional and Metabolic Disorders	2021	View \| Download
Potassium citrate and potassium bicarbonate for distal renal tubular acidosis – first line	Sibnayal (ADV 7103) (fixed dose combination: Potassium bicarbonate; Potassium citrate)	Distal renal tubular acidosis	Endocrine Nutritional and Metabolic Disorders , Nephrology	2020	View \| Download