Sebetralstat for treating acute attacks of hereditary angioedema in people aged 12 and over [ID6284]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 17 December 2025
Donidalorsen for preventing recurrent attacks of hereditary angioedema in people 12 years and over [ID6457]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 18 June 2026
Olezarsen for treating familial chylomicronaemia syndrome [ID6585]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 19 August 2026
Avapritinib for treating inadequately controlled moderate to severe indolent systemic mastocytosis [ID6578]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: 3 September 2026
Rivogenlecleucel for treating haematological non-malignant diseases in children and young people undergoing haploidentical haematopoietic stem cell transplant [ID1496]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Ropeginterferon alfa-2b for treating polycythaemia vera without symptomatic splenomegaly [ID1596]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Daprodustat for treating anaemia in people with chronic kidney disease [ID3987]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Fidanacogene elaparvovec for treating moderately severe to severe haemophilia B [ID4032]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Ravulizumab for untreated thrombotic microangiopathy after a haematopoietic stem cell transplant in people aged 28 days and over [TSID10642][ID6319]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Giroctocogene fitelparvovec for treating moderately severe to severe haemophilia A ID6312Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Rilzabrutinib for treating persistent or chronic immune thrombocytopenia in people aged 12 and over [ID6395]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Inclacumab for preventing recurrent vaso-occlusive crises in sickle cell disease in people 16 years and over [ID6551]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Mozafancogene autotemcel for treating Fanconi anaemia in people 1 to 17 years [ID12203]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Plozasiran for treating familial chylomicronaemia syndrome [ID6593]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Concizumab for treating haemophilia A or B in people 12 years and over with inhibitors [ID6665]Status:In developmentProgramme:Technology appraisal guidanceExpected publication date: TBC