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
VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older	Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) , VX-659 (Bamocaftor-potassium)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
VX-659/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older	Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) , VX-659 (Bamocaftor-potassium)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 years and older	Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
VX-445/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 12 years and older	Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor)	Cystic fibrosis	Genetic Disorders , Respiratory System	2019	View \| Download
Vonicog alfa for Prophylaxis of Haemorrhage in Von Willebrand disease	Vonicog alfa (recombinant von Willebrand factor)	von Willebrand disease	Genetic Disorders , Haematology	2023	View \| Download
Venglustat for treating type 3 Gaucher disease after 1 therapy	Venglustat	Gaucher disease	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2023	View \| Download
Venglustat for treating late onset GM2 gangliosidosis, GM1 gangliosidosis and other ultra-rare disease	Venglustat	GM1 gangliosidosis , GM2 gangliosidosis	Endocrine Nutritional and Metabolic Disorders , Genetic Disorders	2023	View \| Download
Vanzacaftor / tezacaftor / deutivacaftor for the treatment of cystic fibrosis	Deutivacaftor , Tezacaftor (VX-661) , Vanzacaftor	Cystic fibrosis	Genetic Disorders , Respiratory System	2023	View \| Download
Vamorolone for treating Duchenne muscular dystrophy	Vamorolone (VBP15)	Duchenne muscular dystrophy (DMD)	Genetic Disorders , Musculoskeletal System	2021	View \| Download
Troriluzole for spinocerebellar ataxia	Troriluzole	Spinocerebellar ataxia	Genetic Disorders , Neurology	2024	View \| Download