Tafamidis for treating transthyretin amyloidosis with cardiomyopathy (TA696)Product type:GuidanceProgramme:Technology appraisal guidancePublished: 12 May 2021
Sapropterin for treating phenylketonuria [ID1475]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 18 August 2021
Daratumumab in combination for untreated systemic amyloid light-chain amyloidosis [ID3748]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 2 March 2022
Elosulfase alfa for treating mucopolysaccharidosis type IVa (review of HST2) [ID1643]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Nitisinone for treating alkaptonuria [ID2691]Status:ProposedProgramme:Technology appraisal guidanceExpected publication date: TBC
Lumasiran for treating primary hyperoxaluria type 1 [ID3765]Status:ProposedProgramme:Technology appraisal guidanceExpected publication date: TBC
Givosiran for treating acute hepatic porphyria [ID1549]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Eladocagene exuparvovec for treating aromatic L-amino acid decarboxylase deficiency [ID3791]Status:ProposedProgramme:Technology appraisal guidanceExpected publication date: TBC
Lenti-D for treating cerebral adrenoleukodystrophy [ID1284]Status:In developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Burosumab for treating FGF23-related hypophosphataemia in tumour-induced osteomalacia [ID3924 ]Status:ProposedProgramme:Technology appraisal guidanceExpected publication date: TBC
Asfotase alfa for treating paediatric-onset hypophosphatasia (review of HST6) [ID3927]Status:ProposedProgramme:Highly specialised technologies guidanceExpected publication date: TBC