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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

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

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

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

Velmanase alfa for treating alpha-mannosidosis [ID800]

In development [GID-HST10010] 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

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

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

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

Avelumab with axitinib for untreated advanced or metastatic renal cell carcinoma [ID1547]

In development [GID-TA10459] Expected publication date: 08 April 2020

Technology appraisal guidance In development

Veliparib with carboplatin and paclitaxel for untreated epithelial ovarian, fallopian tube or primary peritoneal cancer ID1561

In development [GID-TA10442] Expected publication date: TBC

Technology appraisal guidance In development

Alpelisib in combination with fulvestrant for treating advanced hormone-receptor positive, HER2-negative, PIK3CA-positive breast cancer [ID1412]

In development [GID-TA10398] Expected publication date: 09 December 2020

Technology appraisal guidance In development

Pembrolizumab in combination with chemotherapy for neoadjuvant treatment of triple negative breast cancer [ID1500]

In development [GID-TA10399] Expected publication date: TBC

Technology appraisal guidance In development

Daratumumab in combination for untreated multiple myeloma when stem cell transplant is unsuitable ID1492

In development [GID-TA10441] Expected publication date: TBC

Technology appraisal guidance In development

Andexanet alfa for reversing anticoagulation [ID1101]

In development [GID-TA10440] Expected publication date: 10 June 2020

Technology appraisal guidance In development

Atezolizumab with carboplatin or cisplatin and pemetrexed for untreated advanced non-squamous non-small-cell lung cancer ID1495

In development [GID-TA10395] Expected publication date: 14 October 2020

Technology appraisal guidance In development

Crisaborole for treating mild to moderate atopic dermatitis in people aged 2 years and older (ID1195)

In development [GID-TA10435] Expected publication date: 03 June 2020

Technology appraisal guidance In development

Nivolumab for previously treated locally advanced or metastatic non-squamous non-small-cell lung cancer (CDF review TA484) [ID1572]

In development [GID-TA10513] Expected publication date: TBC

Technology appraisal guidance In development

Nintedanib for treating interstitial lung disease caused by systemic sclerosis (ID1420)

In development [GID-TA10427] Expected publication date: TBC

Technology appraisal guidance In development

Olaparib for maintenance treatment of BRCA mutation-positive metastatic pancreatic cancer after initial platinum-based chemotherapy [ID1518]

In development [GID-TA10504] Expected publication date: TBC

Technology appraisal guidance In development

Carfilzomib with dexamethasone and lenalidomide for treating multiple myeloma after at least 1 previous therapy [ID1493]

In development [GID-TA10510] Expected publication date: TBC

Technology appraisal guidance In development

Acalabrutinib for treating chronic lymphocytic leukaemia ID1613

In development [GID-TA10532] Expected publication date: TBC

Technology appraisal guidance In development

Zynteglo for treating transfusion-dependent beta-thalassaemia ID968

In development [GID-TA10334] Expected publication date: 24 June 2020

Technology appraisal guidance In development

Mexiletine for treating myotonia in adults with non-dystrophic myotonic disorders ID1488

In development [GID-TA10432] Expected publication date: 12 August 2020

Technology appraisal guidance In development

Ticagrelor for preventing cardiovascular events in people with type 2 diabetes and coronary artery disease [ID1514]

In development [GID-TA10429] Expected publication date: TBC

Technology appraisal guidance In development

Neratinib for treating HER2-positive breast cancer after 2 therapies [ID1381]

In development [GID-TA10539] Expected publication date: TBC

Technology appraisal guidance In development

Avapritinib for treating unresectable or metastatic gastrointestinal stromal tumours [ID1626]

In development [GID-TA10523] Expected publication date: 16 December 2020

Technology appraisal guidance In development

Mirvetuximab soravtansine for previously treated platinum-resistant FR-alpha positive ovarian cancer [ID1527]

In development [GID-TA10483] Expected publication date: TBC

Technology appraisal guidance In development

Nivolumab with temozolomide and radiotherapy for newly diagnosed glioblastoma with MGMT methylation [ID1582]

In development [GID-TA10495] Expected publication date: 26 August 2020

Technology appraisal guidance In development

Relatlimab with nivolumab for treating advanced malignant melanoma after immunotherapy in people over 12 [ID1612]

In development [GID-TA10484] Expected publication date: TBC

Technology appraisal guidance In development

Pembrolizumab for treating relapsed or refractory classical Hodgkin's lymphoma after 1 or more multi-agent chemotherapy regimens [ID1557]

In development [GID-TA10485] Expected publication date: 03 February 2021

Technology appraisal guidance In development

Pegargiminase with pemetrexed and cisplatin for untreated advanced malignant pleural mesothelioma [ID1575]

In development [GID-TA10486] Expected publication date: TBC

Technology appraisal guidance In development

Darolutamide for treating non-metastatic hormone-relapsed prostate cancer ID1443

In development [GID-TA10476] Expected publication date: 25 November 2020

Technology appraisal guidance In development

Daratumumab in combination for untreated multiple myeloma when stem cell transplant is suitable [ID1510]

In development [GID-TA10449] Expected publication date: TBC

Technology appraisal guidance In development

Apalutamide with abiraterone acetate and prednisone for treating metastatic hormone-relapsed prostate cancer [ID1480]

In development [GID-TA10465] Expected publication date: TBC

Technology appraisal guidance In development

Encorafenib with binimetinib and cetuximab for previously treated BRAF V600E mutation-positive metastatic colorectal cancer

In development [GID-TA10479] Expected publication date: 22 July 2020

Technology appraisal guidance In development