Somapacitan for treating growth hormone deficiency in people 3 to 17 years (TA1066)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 3 June 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
Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over [ID6160]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
Pegzilarginase for treating arginase-1 deficiency [ID4029]Status:In developmentProgramme:Highly specialised technologies 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:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Eplontersen for treating transthyretin-related amyloidosis cardiomyopathy [TSID12015]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Doxecitine–doxribtimine for treating thymidine kinase 2 deficiency in people of any age ID6484Status:In developmentProgramme:Highly specialised technologies 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
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