Burosumab for treating X-linked hypophosphataemia in adults [ID3822]Status:In developmentProgramme: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
Eplontersen for treating polyneuropathy caused by hereditary transthyretin amyloidosis [ID6337]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 14 August 2024
Leriglitazone for treating andrenoleukodystrophy [ID3903]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
Somapacitan for treating growth hormone deficiency in children [ID6178]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
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