Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over TS ID 10392Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
CAEL-101 with standard care for untreated amyloid light chain amyloidosis TS ID 10666Status: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 TS ID 10612Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Leriglitazone for treating X-linked andrenoleukodystrophy TS ID 9996Status:Awaiting developmentProgramme: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