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.

| 18 Results

Title
Intervention Indication Therapeutic Area Year Actions
Lumacafor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 12 to 23 months Orkambi (fixed dose combination: Lumacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Kaftrio (Elexacaftor/tezacaftor/ivacaftor) and ivacaftor for treating cystic fibrosis in children (2 to 5 years) with at least one F508del mutation in the CFTR gene Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2022 View  |  Download
Ivacaftor for cystic fibrosis in infants Ivacaftor (Kalydeco; VX-770) Cystic fibrosis Genetic Disorders , Respiratory System 2023 View  |  Download
Elexacaftor/tezacaftor/ivacaftor and ivacaftor for treating cystic fibrosis without an F508del mutation and with an ELX/TEZ/IVA-responsive mutation in the CFTR gene in patients aged 6 years and older Ivacaftor (Kalydeco; VX-770) , Kaftrio (fixed dose combination: Elexacaftor; Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2023 View  |  Download
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis homozygous for F508del mutation in patients aged 6 to 11 years old Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation in patients aged 12 years old and older Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Tezacaftor (VX-661) Cystic fibrosis Genetic Disorders , Respiratory System 2020 View  |  Download
Elexacaftor/tezacaftor/ivacaftor (fixed-dose combination) for cystic fibrosis heterozygous for F508del mutation and one minimal function mutation in patients aged 6 to 11 years Elexacaftor (VX-445) , Ivacaftor (Kalydeco; VX-770) , Symkevi (fixed dose combination: Tezacaftor; Ivacaftor) Cystic fibrosis Genetic Disorders , Respiratory System 2019 View  |  Download
Cysteamine bitartrate for cystic fibrosis exacerbations Cysteamine bitartrate (Procysbi; RP103; Lynovex; NM001; Cystadrops; Mercaptamine) Cystic fibrosis Genetic Disorders , Respiratory System 2018 View  |  Download
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