Etranacogene dezaparvovec

• Solution for infusion vials 100 tera genome copies/10ml

Notes

1. NICE TA989: Etranacogene dezaparvovec (Hemgenix) is recommended with managed access as an option for treating moderately severe or severe haemophilia B (congenital factor IX [FIX] deficiency) in adults without anti-FIX antibodies, only if the conditions in the managed access agreement for etranacogene dezaparvovec are followed (July 2024).

Exagamglogene autotemcel

• Dispersion for infusion vials 4million-13million cells/ml

Notes

1. NICE TA1003: Exagamglogene autotemcel (exa-cel) (Casgevy) is recommended with managed access as an option for treating transfusion-dependent beta-thalassaemia in people 12 years and over:
   1. when a haematopoietic stem cell transplant (HSCT) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available
   2. only if the conditions in the managed access agreement for exa-cel are followed (September 2024).
2. NICE TA1044: Exagamglogene autotemcel (exa-cel) (Casgevy) is recommended with managed access as an option for treating sickle cell disease (SCD) in people 12 years and over:
   1. who have:
      1. recurrent vaso-occlusive crises (VOCs) and
      2. a β^S/β^S, β^S/β⁺ or β^S/β⁰ genotype and
   2. when haematopoietic stem cell transplant (HSCT) is suitable, but a human leukocyte antigen-matched related haematopoietic stem cell donor is not available.
   3. It is only recommended for people who have had at least 2 VOCs per year during the 2 previous years and if the conditions in the managed access agreement for exa-cel are followed (February 2025).