Afamelanotide for treating erythropoietic protoporphyria (HST27)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 26 July 2023
Asfotase alfa for treating paediatric-onset hypophosphatasia (HST23)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 1 March 2023
Atidarsagene autotemcel for treating metachromatic leukodystrophy (HST18)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 March 2022
Avalglucosidase alfa for treating Pompe disease (TA821)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 24 August 2022
Burosumab for treating FGF23-related hypophosphataemia in tumour-induced osteomalacia [ID3924 ]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Burosumab for treating X-linked hypophosphataemia in adults [ID3822]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
CAEL-101 with standard care for untreated amyloid light chain amyloidosis TS ID 10666Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 August 2023
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease in people 12 to 17 years TS ID 11887Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Daratumumab in combination for treating newly diagnosed systemic amyloid light-chain amyloidosis (TA959)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 March 2024
Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over TS ID 10392Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency (HST26)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 19 April 2023
Eliglustat for treating type 1 Gaucher disease (HST5)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 28 June 2017
Elosulfase alfa for treating mucopolysaccharidosis type 4A (HST19)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 20 April 2022
Eplontersen for treating polyneuropathy caused by hereditary transthyretin amyloidosis [ID6337]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 14 August 2024
Givosiran for treating acute hepatic porphyria (HST16)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 24 November 2021
Human growth hormone (somatropin) in adults with growth hormone deficiency (TA64)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 August 2003
Inotersen for treating hereditary transthyretin amyloidosis (HST9)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 22 May 2019
Leriglitazone for treating andrenoleukodystrophy [ID3903]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Leriglitazone for treating X-linked andrenoleukodystrophy TS ID 9996Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Nitisinone for treating alkaptonuria [ID2691]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Patisiran for treating hereditary transthyretin amyloidosis (HST10)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 14 August 2019
Pegzilarginase for treating arginase-1 deficiency [ID4029]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 22 September 2021
Slow-release potassium bicarbonate–potassium citrate for treating distal renal tubular acidosis (terminated appraisal) (TA838)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 2 November 2022
Somapacitan for treating growth hormone deficiency in children [ID6178]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Somatrogon for treating growth disturbance in children and young people aged 3 years and over (TA863)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 1 February 2023
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA696)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 12 May 2021
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy [ID6327]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 12 June 2024
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Velmanase alfa for treating alpha-mannosidosis (HST29)Product type:GuidanceProgramme:Highly specialised technologies guidancePublished: 13 December 2023
Venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy TS ID 10612Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Vutrisiran for treating hereditary transthyretin-related amyloidosis (TA868)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 February 2023