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.

| 61 Results

Title
Intervention Indication Therapeutic Area Year Actions
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
Emapalumab for Primary Haemophagocytic Lymphohistiocytosis Emapalumab (NI-0501; Gamifant) Primary haemophagocytic lymphohistiocytosis (HLH) Genetic Disorders , Immunology 2018 View  |  Download
Fordadistrogene movaparvovec for Duchenne muscular dystrophy Fordadistrogene movaparvovec Duchenne muscular dystrophy (DMD) Genetic Disorders , Musculoskeletal System 2022 View  |  Download
Inclisiran for primary hypercholesterolaemia and mixed dyslipidaemia Inclisiran (ALN-PCSsc; ALN-60212) Heterozygous familial hypercholesterolaemia (HeFH) , Primary hypercholesterolaemia Endocrine Nutritional and Metabolic Disorders , Genetic Disorders 2019 View  |  Download
Lanadelumab for prevention of attacks in adults and adolescents with Type I and Type II hereditary angioedema Lanadelumab (SHP643; TAK-743; Takhzyro) Hereditary angioedema (HAE) Genetic Disorders , Immunology 2017 View  |  Download
Lenadogene Nolparvovec (GS-010) for Vision Loss from Leber's Hereditary Optic Neuropathy Due to Mutation of the ND4 Gene Lenadogene nolparvovec (Lumevoq; GS-010) Leber's hereditary optic neuropathy (LHON) Genetic Disorders , Ophthalmology 2017 View  |  Download
Leniolisib for previously untreated activated phosphoinositide 3-kinase delta syndrome Leniolisib Activated PI3K delta syndrome Genetic Disorders 2022 View  |  Download
Lenti‐D for adrenoleukodystrophy in boys Elivaldogene tavalentivec (Lenti-D) Adrenoleukodystrophy (ALD) Genetic Disorders , Neurology 2017 View  |  Download
Leriglitazone for X-linked andrenoleukodystrophy Leriglitazone (MIN-102) X-linked adrenoleukodystrophy (X-ALD) Endocrine Nutritional and Metabolic Disorders , Genetic Disorders , Neurology 2020 View  |  Download
Lonafarnib for Hutchinson-Gilford Progeria syndrome and progeroid laminopathies Lonafarnib (Sarasar; EBP994; SCH 66336; Zokinvy) Progeria Genetic Disorders 2020 View  |  Download
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