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NICE is unable to make a recommendation on idecabtagene vicleucel (Abecma) for treating relapsed and refractory multiple myeloma after 3 or more treatments in adults. This is because BMS did not provide an evidence submission. We will review this decision if the company decides to make a submission.
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Sections for TA936
Ravulizumab for treating generalised myasthenia gravis (terminated appraisal) (TA940)
NICE is unable to make a recommendation on ravulizumab (Ultomiris) for treating generalised myasthenia gravis in adults. This is because Alexion Pharma UK withdrew its evidence submission. We will review this decision if the company decides to make a submission.
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Sections for TA940
NICE is unable to make a recommendation on ravulizumab (Ultomiris) for treating AQP4 antibody-positive neuromyelitis optica spectrum disorder in adults. This is because Alexion Pharma UK withdrew its evidence submission. We will review this decision if the company decides to make a submission.
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Sections for TA941
NICE is unable to make a recommendation on dupilumab (Dupixent) for treating eosinophilic oesophagitis in people 12 years and over. This is because Sanofi did not provide an evidence submission. We will review this decision if the company decides to make a submission.
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Sections for TA938
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.
Evidence-based recommendations on voretigene neparvovec (Luxturna) for RPE65-mediated inherited retinal dystrophies in people with vision loss caused by inherited retinal dystrophy from confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.
Cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2 (HST12)
Evidence-based recommendations on cerliponase alfa (Brineura) for treating neuronal ceroid lipofuscinosis type 2 in children.
Volanesorsen for treating familial chylomicronaemia syndrome (HST13)
Evidence-based recommendations on volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome in adults.
Evidence-based recommendations on metreleptin (Myalepta) for treating lipodystrophy in children and adults.
Onasemnogene abeparvovec for treating spinal muscular atrophy (HST15)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating spinal muscular atrophy in babies.
Eculizumab for treating atypical haemolytic uraemic syndrome (HST1)
Evidence-based recommendations on eculizumab (Soliris) for treating atypical haemolytic uraemic syndrome in adults and children.
Evidence-based recommendations on ataluren (Translarna) for Duchenne muscular dystrophy with a nonsense mutation in the dystrophin gene in people 2 years and over who can walk.
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)
Evidence-based recommendations on asfotase alfa (Strensiq) for treating paediatric-onset hypophosphatasia in babies, children, young people and adults.
Onasemnogene abeparvovec for treating presymptomatic spinal muscular atrophy (HST24)
Evidence-based recommendations on onasemnogene abeparvovec (Zolgensma) for treating presymptomatic spinal muscular atrophy in babies aged 12 months and under.
Evidence-based recommendations on lumasiran (Oxlumo) for primary hyperoxaluria type 1 in people of all ages.
Evidence-based recommendations on givosiran (Givlaari) for treating acute hepatic porphyria in adults and young people aged 12 and over.
Odevixibat for treating progressive familial intrahepatic cholestasis (HST17)
Evidence-based recommendations on odevixibat (Bylvay) for treating progressive familial intrahepatic cholestasis in people 6 months and older.
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)
Evidence-based recommendations on atidarsagene autotemcel (Libmeldy) for treating metachromatic leukodystrophy in children.
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)
Evidence-based recommendations on elosulfase alfa (Vimizim) for treating mucopolysaccharidosis type 4A in people of all ages.
Evidence-based recommendations on selumetinib (Koselugo) for treating symptomatic and inoperable plexiform neurofibromas associated with type 1 neurofibromatosis in children aged 3 and over.
Afamelanotide for treating erythropoietic protoporphyria (HST27)
Evidence-based recommendations on afamelanotide (Scenesse) for treating erythropoietic protoporphyria in adults.
Evidence-based recommendations on velmanase alfa (Lamzede) for treating alpha-mannosidosis in people under 18 years and in people who turn 18 while on treatment.
Evidence-based recommendations on migalastat (Galafold) for treating Fabry disease in people over 16.
Evidence-based recommendations on eliglustat (Cerdelga) for treating type 1 Gaucher disease in adults.
Strimvelis for treating adenosine deaminase deficiency–severe combined immunodeficiency (HST7)
Evidence-based recommendation on Strimvelis for adenosine deaminase deficiency–severe combined immunodeficiency.
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.
Evidence-based recommendations on tafasitamab (Minjuvi) with lenolidomide for relapsed or refractory diffuse large B-cell lymphoma in adults who cannot have an autologous stem cell transplant.
Evidence-based recommendations on olaparib (Lynparza) for adjuvant treatment of BRCA mutation-positive HER2-negative high-risk early breast cancer after chemotherapy in adults.
Difelikefalin for treating pruritus in people having haemodialysis (TA890)
Evidence-based recommendations on difelikefalin (Kapruvia) for pruritus in adults with chronic kidney disease having haemodialysis.
Ibrutinib with venetoclax for untreated chronic lymphocytic leukaemia (TA891)
Evidence-based recommendations on ibrutinib (Imbruvica) with venetoclax (Venclyxto) for untreated chronic lymphocytic leukaemia in adults.
Evidence-based recommendations on risdiplam (Evrysdi) for 5q spinal muscular atrophy (SMA) in people of all ages.
Deucravacitinib for treating moderate to severe plaque psoriasis (TA907)
Evidence-based recommendations on deucravacitinib for treating moderate to severe plaque psoriasis in adults.
Evidence-based recommendations on olaparib (Lynparza) for maintenance treatment of relapsed, platinum-sensitive ovarian, fallopian tube or peritoneal cancer after 2 or more courses of platinum-based chemotherapy in adults.
Selpercatinib for untreated RET fusion-positive advanced non-small-cell lung cancer (TA911)
Evidence-based recommendations on selpercatinib (Retsevmo) for untreated RET fusion-positive advanced non-small-cell lung cancer in adults.
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)
Evidence-based recommendations on cipaglucosidase alfa (Pombiliti) with miglustat (Opfolda) for treating late-onset Pompe disease in adults.
Mavacamten for treating symptomatic obstructive hypertrophic cardiomyopathy (TA913)
Evidence-based recommendations on mavacamten (Camzyos) for symptomatic obstructive hypertrophic cardiomyopathy.
Evidence-based recommendations on pembrolizumab (Keytruda) for previously treated endometrial, biliary, colorectal, gastric or small intestine cancer with high microsatellite instability or mismatch repair deficiency in adults.
Evidence-based recommendations on pegunigalsidase alfa (Elfabrio) for Fabry disease (also known as alpha-galactosidase deficiency) in adults.
Evidence-based recommendations on rimegepant (Vydura) for the acute treatment of migraine in adults.
Evidence-based recommendations on ruxolitinib (Jakavi) for polycythaemia vera in adults.
Evidence-based recommendations on baricitinib (Olumiant) for treating severe alopecia areata in adults.
Evidence-based recommendations on cabozantinib (Cabometyx) for previously treated advanced differentiated thyroid cancer unsuitable for or refractory to radioactive iodine, in adults.
Evidence-based recommendations on empagliflozin (Jardiance) for treating chronic heart failure with preserved or mildly reduced ejection fraction in adults.
Evidence-based recommendations on axicabtagene ciloleucel (Yescarta) for treating relapsed or refractory diffuse large B-cell lymphoma after first-line chemoimmunotherapy.
Evidence-based recommendations on bulevirtide (Hepcludex) for chronic hepatitis D in adults.
Daratumumab with bortezomib and dexamethasone for previously treated multiple myeloma (TA897)
Evidence-based recommendations on daratumumab (Darzalex) with bortezomib and dexamethasone for previously treated multiple myeloma in adults.
Evidence-based recommendations on dabrafenib (Tafinlar) plus trametinib (Mekinist) for BRAF V600 mutation-positive advanced non-small-cell lung cancer in adults.
Evidence-based recommendations on dapagliflozin (Forxiga) for treating chronic heart failure with preserved or mildly reduced ejection fraction in adults.
Evidence-based recommendations on darolutamide (Nubeqa) with androgen deprivation therapy and docetaxel for hormone-sensitive metastatic prostate cancer in adults.
Evidence-based recommendations on pembrolizumab (Keytruda) with lenvatinib (Lenvima) for treating advanced or recurrent endometrial cancer in adults.