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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

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

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

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

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

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

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

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

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

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

Intrathecal idursulfase for treating Mucopolysaccharidosis type II ID1223

Proposed [GID-HST10019] Expected publication date: TBC

Highly specialised technologies guidance Proposed

Metreleptin for treating lipodystrophy (ID861)

In development [GID-HST10011] Expected publication date: TBC

Highly specialised technologies guidance In development

Afamelanotide for treating erythropoietic protoporphyria [ID927]

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

Highly specialised technologies guidance In development

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

Proposed [GID-HST10020] Expected publication date: TBC

Highly specialised technologies guidance Proposed

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

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

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

In development [GID-HST10027] 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

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

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

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

Lenalidomide for previously treated follicular lymphoma and marginal zone lymphoma [ID1374]

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

Technology appraisal guidance In development

Denosumab for preventing skeletal-related events in multiple myeloma (terminated appraisal) (TA549)

NICE is unable to make a recommendation about the use in the NHS of denosumab for preventing skeletal-related events in multiple myeloma because no evidence

Technology appraisal guidance Published December 2018

Decitabine for untreated acute myeloid leukaemia (terminated appraisal) (TA548)

NICE is unable to make a recommendation about the use in the NHS of decitabine for untreated acute myeloid leukaemia because no evidence submission was

Technology appraisal guidance Published December 2018

Lenalidomide with bortezomib and dexamethasone for untreated multiple myeloma (terminated appraisal) (TA603)

NICE is unable to make a recommendation about the use in the NHS of lenalidomide (Revlimid) with bortezomib and dexamethasone for untreated multiple myeloma

Technology appraisal guidance Published September 2019

Pomalidomide with bortezomib and dexamethasone for treating relapsed or refractory multiple myeloma (terminated appraisal) (TA602)

NICE is unable to make a recommendation about the use in the NHS of pomalidomide (Imnovid) with bortezomib and dexamethasone for relapsed or refractory myeloma

Technology appraisal guidance Published September 2019

Bezlotoxumab for preventing recurrent Clostridium difficile infection (terminated appraisal) (TA601)

NICE is unable to make a recommendation about the use in the NHS of bezlotoxumab (Zinplava) for preventing recurrent Clostridium difficile infection in adults

Technology appraisal guidance Published September 2019

Brentuximab vedotin for untreated advanced Hodgkin lymphoma (terminated appraisal) (TA594)

NICE is unable to make a recommendation about the use in the NHS of brentuximab vedotin (Adcetris) for untreated advanced Hodgkin lymphoma in adults

Technology appraisal guidance Published August 2019

Cabozantinib for previously treated advanced hepatocellular carcinoma (terminated appraisal) (TA582)

NICE is unable to make a recommendation about the use in the NHS of cabozantinib (Cometriq) for previously treated advanced hepatocellular carcinoma in adults

Technology appraisal guidance Published May 2019

Ixekizumab for treating moderate to severe plaque psoriasis (TA442)

Evidence-based recommendations on ixekizumab (Taltz) for moderate to severe plaque psoriasis in adults

Technology appraisal guidance Published April 2017

Tenofovir alafenamide for treating chronic hepatitis B (terminated appraisal) (TA435)

NICE was unable to make a recommendation about the use in the NHS of tenofovir alafenamide for treating chronic hepatitis B because no evidence submission was

Technology appraisal guidance Published March 2017

Elotuzumab for previously treated multiple myeloma (terminated appraisal) (TA434)

NICE was unable to make a recommendation about the use in the NHS of elotuzumab for previously treated multiple myeloma because no evidence submission was

Technology appraisal guidance Published March 2017

Guidance on the use of drugs for early thrombolysis in the treatment of acute myocardial infarction (TA52)

Evidence-based recommendations on streptokinase (Streptase), alteplase (Actilyse), reteplase (Rapilysin) and tenecteplase (Metalyse) for early thrombolysis in

Technology appraisal guidance Published October 2002

Guidance on the use of ultrasound locating devices for placing central venous catheters (TA49)

Evidence-based recommendations on ultrasound locating devices for placing central venous catheters into the internal jugular vein

Technology appraisal guidance Published October 2002

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

Denosumab for the prevention of skeletal-related events in adults with bone metastases from solid tumours (TA265)

Evidence-based recommendations on denosumab (XGEVA) for the prevention of skeletal-related events in adults with bone metastases from solid tumours

Technology appraisal guidance Published October 2012

Abatacept for treating psoriatic arthritis after DMARDs (terminated appraisal) (TA568)

NICE is unable to make a recommendation on abatacept (Orencia) for treating psoriatic arthritis after DMARDs in adults because of no evidence submission from

Technology appraisal guidance Published March 2019

Dabrafenib with trametinib for treating advanced metastatic BRAF V600E mutation-positive non-small-cell lung cancer (terminated appraisal) (TA564)

NICE is unable to make a recommendation about the use in the NHS of dabrafenib with trametinib for treating advanced metastatic BRAF V600E mutation-positive

Technology appraisal guidance Published February 2019

Pembrolizumab for treating recurrent or metastatic squamous cell carcinoma of the head and neck after platinum-based chemotherapy (terminated appraisal) (TA570)

NICE is unable to make a recommendation about the use in the NHS of pembrolizumab (Keytruda) for treating recurrent or metastatic squamous cell carcinoma of the

Technology appraisal guidance Published March 2019

Loxapine inhalation for treating acute agitation and disturbed behaviours associated with schizophrenia and bipolar disorder (terminated appraisal) (TA286)

NICE is unable to recommend the use in the NHS of loxapine inhalation for treating acute agitation and disturbed behaviours associated with

Technology appraisal guidance Published May 2013

Colistimethate sodium and tobramycin dry powders for inhalation for treating pseudomonas lung infection in cystic fibrosis (TA276)

Evidence-based recommendations on colistimethate sodium and tobramycin dry powders for inhalation for pseudomonas lung infection in cystic fibrosis

Technology appraisal guidance Published March 2013

Percutaneous vertebroplasty and percutaneous balloon kyphoplasty for treating osteoporotic vertebral compression fractures (TA279)

Evidence-based recommendations on percutaneous vertebroplasty and percutaneous balloon kyphoplasty for osteoporotic vertebral compression fractures

Technology appraisal guidance Published April 2013

Methylnaltrexone for treating opioid-induced bowel dysfunction in people with advanced illness receiving palliative care (terminated appraisal) (TA277)

NICE is unable to recommend the use in the NHS of methylnaltrexone for treating opioid-induced bowel dysfunction in people with advanced illness...

Technology appraisal guidance Published March 2013

Afatinib for treating advanced squamous non-small-cell lung cancer after platinum-based chemotherapy (terminated appraisal) (TA444)

NICE is unable to make a recommendation about the use in the NHS of afatinib for advanced squamous non-small-cell lung cancer after platinum-based chemotherapy

Technology appraisal guidance Published May 2017