OTL-101 for treating adenosine deaminase deficiency–severe combined immunodeficiency [ID1152]Status:Awaiting developmentProgramme:Highly specialised technologies guidanceExpected publication date: TBC
Treosulfan with fludarabine for non-malignant disease before allogeneic stem cell transplant [ID1540]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Valoctocogene roxaparvovec for treating severe haemophilia A [ID3806]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Luspatercept for treating anaemia in non-transfusion dependent beta-thalassaemia [ID3870]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Concizumab for preventing bleeding episodes in haemophilia A or haemophilia B [ID5099]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Apadamtase alfa for preventing and treating congenital thrombotic thrombocytopenic purpura caused by ADAMTS-13 deficiency [ID6192]Status:Awaiting 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]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Benralizumab for treating hypereosinophilic syndrome in people 12 years and over [ID6322]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Dupilumab for treating eosinophilic oesophagitis in children 1 to 11 years [ID6492]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Rilzabrutinib for treating persistent or chronic immune thrombocytopenia in people aged 12 and over [ID6395]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Subcutaneous efgartigmod with recombinant human hyaluronidase PH20 for treating primary immune thrombocytopenia [TSID11977]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
MIM8 for preventing bleeding episodes in haemophilia A in people 1 year and over [ID6400]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Donidalorsen for preventing hereditary angioedema attacks in people 12 years and over [ID6457]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Budesonide oral suspension for maintaining remission of eosinophilic oesophagitis in people 2 years and over [ID6486]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Olezarsen for treating familial chylomicronaemia syndrome [TSID11993]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Inclacumab for preventing recurrent vaso-occlusive crises in sickle cell disease in people 16 years and over [TSID12144]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC
Marstacimab for treating severe haemophilia A or B in people 12 years and over with inhibitors to factor-replacement therapy [TSID12143]Status:Awaiting developmentProgramme:Technology appraisal guidanceExpected publication date: TBC