Eplontersen for treating hereditary transthyretin-related amyloidosis (TA1020)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 November 2024
Burosumab for treating X-linked hypophosphataemia in adults (TA993)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 7 August 2024
Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA984)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 19 June 2024
Daratumumab in combination for treating newly diagnosed systemic amyloid light-chain amyloidosis (TA959)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 27 March 2024
Pegunigalsidase alfa for treating Fabry disease (TA915)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 4 October 2023
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease (TA912)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 August 2023
Mitapivat for treating pyruvate kinase deficiency (terminated appraisal) (TA867)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 16 February 2023
Vutrisiran for treating hereditary transthyretin-related amyloidosis (TA868)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 15 February 2023
Slow-release potassium bicarbonate–potassium citrate for treating distal renal tubular acidosis (terminated appraisal) (TA838)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 2 November 2022
Avalglucosidase alfa for treating Pompe disease (TA821)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 24 August 2022
Sodium zirconium cyclosilicate for treating hyperkalaemia (TA599)Product type:GuidanceProgramme:Technology appraisal guidanceLast updated: 24 January 2022Published: 4 September 2019
Sapropterin for treating hyperphenylalaninaemia in phenylketonuria (TA729)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 22 September 2021
Patiromer for treating hyperkalaemia (TA623)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 13 February 2020
Somapacitan for treating growth hormone deficiency in people 3 to 17 years [ID6178]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 29 May 2025
Sodium zirconium cyclosilicate for treating hyperkalaemia (partial review of TA599) [ID6439]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 17 December 2025
Vutrisiran for treating transthyretin-related amyloidosis cardiomyopathy [ID6470]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 28 January 2026
Nitisinone for treating alkaptonuria [ID2691]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Burosumab for treating FGF23-related hypophosphataemia in tumour-induced osteomalacia [ID3924 ]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Leriglitazone for treating andrenoleukodystrophy [ID3903]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [TSID10392]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
CAEL-101 with standard care for untreated amyloid light chain amyloidosis [ID6210]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy [ID6295]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Leriglitazone for treating X-linked andrenoleukodystrophy [TSID9996]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Cipaglucosidase alfa with miglustat for treating late-onset Pompe disease in people 12 to 17 years [TSID11887]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
Triheptanoin for treating long-chain fatty acid oxidation disorders [ID3891]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Venglustat for treating gangliosidoses in people 2 years and over ID 6358Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age who had symptoms before 13 years [TSID12046]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Govorestat for treating classic galactosemia in people 2 to 65 years [TSID12071]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Prolonged-release diazoxide choline for treating Prader-Willi syndrome [ID1389]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC
UX111 for treating mucopolysaccharidosis type IIIA [ID6540]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Elamipretide for treating Barth syndrome in people of any age [ID6545]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Veverimer for treating metabolic acidosis in chronic kidney disease [ID3832]Status:Topic selectionProgramme:Technology appraisal guidanceExpected publication date: TBC