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Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)

Evidence-based recommendations on culizumab (Soliris) for treating atypical haemolytic uraemic syndrome (aHUS)

Highly specialised technologies guidance Published January 2015

Inotersen for treating hereditary transthyretin amyloidosis (HST9)

Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis

Highly specialised technologies guidance Published May 2019

Burosumab for treating X-linked hypophosphataemia in children and young people (HST8)

Evidence-based recommendations on burosumab (Crysvita) for treating X-linked hypophosphataemia (XLH) in children and young people

Highly specialised technologies guidance Published October 2018

Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)

Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID)

Highly specialised technologies guidance Published February 2018

Asfotase alfa for treating paediatric-onset hypophosphatasia (HST6)

Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in adults and children

Highly specialised technologies guidance Published August 2017

Eliglustat for treating type 1 Gaucher disease (HST5)

Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults

Highly specialised technologies guidance Published June 2017

Migalastat for treating Fabry disease (HST4)

Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16

Highly specialised technologies guidance Published February 2017

Ataluren for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene (HST3)

Evidence-based recommendations on ataluren (Translarna) for treating Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene..

Highly specialised technologies guidance Published July 2016

Elosulfase alfa for treating mucopolysaccharidosis type IVa (HST2)

Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type IVa (MPS IVA)

Highly specialised technologies guidance Published December 2015

Voretigene neparvovec for treating inherited retinal dystrophies caused by RPE65 gene mutations (HST11)

Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies (confirmed biallelic RPE65 gene mutations)

Highly specialised technologies guidance Published October 2019

Patisiran for treating hereditary transthyretin amyloidosis (HST10)

Evidence-based recommendations on patisiran (Onpattro) for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy

Highly specialised technologies guidance Published August 2019

Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)

Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 (CLN2) in children

Highly specialised technologies guidance Published November 2019

VTS-270 for treating Niemann-Pick type C1 (ID1267)

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Onasemnogene abeparvovec for treating spinal muscular atrophy type 1 [ID1473]

In development [GID-HST10026] Expected publication date: 08 September 2020

Highly specialised technologies guidance In development

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] Expected publication date: TBC

Highly specialised technologies guidance In development

OTL-200 for treating metachromatic leukodystrophy [ID1666]

Proposed [GID-HST10028] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]

In development [GID-HST10027] Expected publication date: 11 November 2020

Highly specialised technologies guidance In development

OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]

Proposed [GID-HST10022] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Ravulizumab for treating paroxysmal nocturnal haemoglobinuria ID1457

Proposed [GID-HST10023] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Afamelanotide for treating erythropoietic protoporphyria [ID927]

In development [GID-HST10009] Expected publication date: TBC

Highly specialised technologies guidance In development

Lysosomal acid lipase deficiency - sebelipase alfa [ID737]

In development [GID-LYSOSOMALACIDLIPASEDEFICIENCYSEBELIPASEALFAID737] Expected publication date: TBC

Highly specialised technologies guidance In development

Human alpha1-proteinase inhibitor for treating emphysema ID856

In development [GID-HST10017] Expected publication date: TBC

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]

In development [GID-HST10015] Expected publication date: 03 June 2020

Highly specialised technologies guidance In development

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: 13 January 2021

Highly specialised technologies guidance In development

Volanesorsen for treating familial chylomicronaemia syndrome [ID1326]: evaluation consultation

We are listening to your views on this highly specialised technology. Comments close 27 January 2020.

Highly specialised technologies guidance In consultation

Drisapersen for the first-line treatment of Duchenne's muscular dystrophy [ID911]

In development [GID-HST10004] Expected publication date: TBC

Highly specialised technologies guidance In development

Eteplirsen for treating Duchenne muscular dystrophy [ID1003]

In development [GID-HST10007] Expected publication date: TBC

Highly specialised technologies guidance In development

Blinatumomab for treating Philadelphia-chromosome-positive relapsed or refractory acute lymphoblastic leukaemia [ID1008]

In development [GID-TA10119] Expected publication date: 01 July 2020

Technology appraisal guidance In development

Olaparib for treating BRCA 1 or 2 mutated metastatic breast cancer after prior chemotherapy [ID1382]

In development [GID-TA10342] Expected publication date: 01 July 2020

Technology appraisal guidance In development

Pembrolizumab with axitinib for untreated metastatic renal cell carcinoma [ID1426]

In development [GID-TA10331] Expected publication date: 01 April 2020

Technology appraisal guidance In development

Lenalidomide for previously treated follicular lymphoma [ID1374]

In development [GID-TA10323] Expected publication date: 01 April 2020

Technology appraisal guidance In development

Venetoclax for treating chronic lymphocytic leukaemia (TA487)

Evidence-based recommendations on venetoclax (Venclyxto) for treating chronic lymphocytic leukaemia in adults

Technology appraisal guidance Published November 2017

Aflibercept for treating choroidal neovascularisation (TA486)

Evidence-based recommendations on aflibercept (Eylea) for treating myopic choroidal neovascularisation in adults

Technology appraisal guidance Published November 2017

Sarilumab for moderate to severe rheumatoid arthritis (TA485)

Evidence-based recommendations on sarilumab (Kevzara) for treating moderate to severe rheumatoid arthritis in adults

Technology appraisal guidance Published November 2017

Nivolumab for previously treated non-squamous non-small-cell lung cancer (TA484)

Evidence-based recommendations on nivolumab (Opdivo) for locally advanced or metastatic (secondary) non-squamous non-small-cell lung cancer in adults after

Technology appraisal guidance Published November 2017

Nivolumab for previously treated squamous non-small-cell lung cancer (TA483)

Evidence-based recommendations on nivolumab (Opdivo) for locally advanced or metastatic squamous non-small-cell lung cancer (NSCLC) in adults after chemotherapy

Technology appraisal guidance Published November 2017

Immunosuppressive therapy for kidney transplant in children and young people (TA482)

Evidence-based recommendations on immunosuppressive therapy for kidney transplant in children and young people

Technology appraisal guidance Published October 2017

Immunosuppressive therapy for kidney transplant in adults (TA481)

Evidence-based recommendations on immunosuppressive therapies for preventing kidney rejection in adults

Technology appraisal guidance Published October 2017

Tofacitinib for moderate to severe rheumatoid arthritis (TA480)

Evidence-based recommendations on tofacitinib (Xeljanz) for treating moderate to severe rheumatoid arthritis (rheumatism) in adults

Technology appraisal guidance Published October 2017

Reslizumab for treating severe eosinophilic asthma (TA479)

Evidence-based recommendations on reslizumab (Cinqaero) for treating severe eosinophilic asthma in adults

Technology appraisal guidance Published October 2017

Brentuximab vedotin for treating relapsed or refractory systemic anaplastic large cell lymphoma (TA478)

Evidence-based recommendations on brentuximab vedotin (Adcetris) for treating relapsed or refractory systemic anaplastic large cell lymphoma in adults

Technology appraisal guidance Published October 2017

Autologous chondrocyte implantation for treating symptomatic articular cartilage defects of the knee (TA477)

Evidence-based recommendations on autologous chondrocyte implantation (ACI) in people with symptomatic articular cartilage defects of the knee

Technology appraisal guidance Published October 2017

Paclitaxel as albumin-bound nanoparticles with gemcitabine for untreated metastatic pancreatic cancer (TA476)

Evidence-based recommendations on paclitaxel as albumin-bound nanoparticles (nab-paclitaxel) for untreated metastatic pancreatic cancer in adults

Technology appraisal guidance Published September 2017

Dimethyl fumarate for treating moderate to severe plaque psoriasis (TA475)

Evidence-based recommendations on dimethyl fumarate (Skilarence) for treating moderate to severe plaque psoriasis in adults

Technology appraisal guidance Published September 2017

Sorafenib for treating advanced hepatocellular carcinoma (TA474)

Evidence-based recommendations on sorafenib (Nexavar) for treating advanced hepatocellular carcinoma (liver cancer) in adults

Technology appraisal guidance Published September 2017

Cetuximab for treating recurrent or metastatic squamous cell cancer of the head and neck (TA473)

Evidence-based recommendations on cetuximab (Erbitux) for treating recurrent or metastatic squamous cell cancer of the head and neck in adults

Technology appraisal guidance Published August 2017

Obinutuzumab with bendamustine for treating follicular lymphoma refractory to rituximab (TA472)

Evidence-based recommendations on obinutuzumab (Gazyvaro) with bendamustine for adults with follicular lymphoma unresponsive to treatment with rituximab

Technology appraisal guidance Published August 2017

Eluxadoline for treating irritable bowel syndrome with diarrhoea (TA471)

Evidence-based recommendations on eluxadoline (Truberzi) for treating irritable bowel syndrome (IBS) with diarrhoea in adults

Technology appraisal guidance Published August 2017

Guidance on the use of glycoprotein IIb/IIIa inhibitors in the treatment of acute coronary syndromes (TA47)

Evidence-based recommendations on glycoprotein IIb/IIIa inhibitors(abciximab [ReoPro], eptifibatide [Integrilin], tirofiban [Aggrastat]) for acute coronary

Technology appraisal guidance Published September 2002 Last updated March 2010