Tafamidis for treating transthyretin amyloidosis with cardiomyopathy [ID6327]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 12 June 2024
Eplontersen for treating polyneuropathy caused by hereditary transthyretin amyloidosis [ID6337]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 14 August 2024
Burosumab for treating X-linked hypophosphataemia in adults [ID3822]Status:In developmentProgramme: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
Somapacitan for treating growth hormone deficiency in children [ID6178]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Pegzilarginase for treating arginase-1 deficiency [ID4029]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC