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.

| 91 Results

Intervention Indication Therapeutic Area Year Actions
A4250 for progressive familial intrahepatic cholestasis Odevixibat (A4250) Progressive familial intrahepatic cholestasis (PFIC) Genetic Disorders , Hepatology 2017 View  |  Download
Alpelisib for treating PIK3CA-related overgrowth spectrum Alpelisib (BYL719; Piqray) PIK3CA-related overgrowth spectrum (PROS) Genetic Disorders 2023 View  |  Download
Arimoclomol for Niemann-Pick Disease type C - add-on therapy Arimoclomol (BRX-345) Niemann-Pick Disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders , Neurology 2017 View  |  Download
Ataluren for Duchenne Muscular Dystrophy with nonsense mutation Ataluren (Translarna; PTC 124; PTC-124; PTC124) Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2022 View  |  Download
ATB200/AT2221 for Late onset Pompe disease in adults and adolescents over 12 years – first line ATB200/AT2221 Pompe disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2020 View  |  Download
Avalglucosidase alfa for late-onset Pompe disease Avalglucosidase alfa (neoGAA; GZ-402666) Pompe disease Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
AVXS-101 for spinal muscular atrophy Onasemnogene abeparvovec (Zolgensma; AVXS-101; scAAV9.CB.SMN) Spinal muscular atrophy (SMA) Genetic Disorders , Musculoskeletal System 2018 View  |  Download
Bardoxolone Methyl for the treatment of chronic kidney disease (CKD) caused by Alport Syndrome Bardoxolone Methyl Alport Syndrome , Chronic kidney disease (CKD) Genetic Disorders , Nephrology 2022 View  |  Download
BCX7353 for prevention of acute attacks of angioedema in hereditary angioedema BCX7353 (Berotralstat; ORLADEYO) Hereditary angioedema (HAE) Genetic Disorders , Immunology 2019 View  |  Download
Belzutifan for von Hippel-Lindau disease-associated clear cell renal cell carcinoma – first line Belzutifan (PT2977; MK-6482) Renal cell carcinoma (RCC) Genetic Disorders , Oncology , Renal Cancer 2021 View  |  Download
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