Exagamglogene autotemcel Medicine 07 June 2022 Extended indication - Sickle cell disease (SCD) - Beta-thalassemia, transfusion-dependent (TDT) Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Exagamglogene autotemcel Domain Cardiovascular diseases Reason of inclusion New medicine (specialité) Main indication Other non-oncological hematological medications Extended indication - Sickle cell disease (SCD) - Beta-thalassemia, transfusion-dependent (TDT) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Vertex Portfolio holder Vertex Mechanism of action Autologous modified cell therapy Route of administration Intravenous Therapeutical formulation Intravenous drip Budgetting framework Intermural (MSZ) Centre of expertise AUMC; UMCU/Creveld Kliniek; LUMC; ErasmusMC Additional remarks Substance a.k.a. CTX001 Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity New medicine with Priority Medicines (PRIME) ATMP Yes Submission date December 2022 Expected Registration September 2023 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options For SCD: Hydroxyurea/hydroxycarbamide and red cell transfusions (top up / exchange) For TDT: Regular blood transfusions and iron chelation therapy, luspatercept. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References NCT03655678 Additional remarks Administered by IV infusion following myeloablative conditioning with busulfan. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References hematologienederland.nl Additional remarks In Nederland zijn ongeveer 100-200 patiënten bekend. De ziekte komt evenveel voor bij mannen als bij vrouwen. Expected cost per patient per year Cost € < 2,000,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References https://www.fool.com/investing/2021/07/23/3-things-about-crispr-therapeutics-smart-investors/ Additional remarks De prijs in Nederland is nog niet bekend. Prijs inschattingen in de Verenigde Staten lopen tot twee miljoen dollar per patiënt. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References adisinsight; fabrikant Additional remarks Other information Additional remarks Frangoul et al 2021. N Engl J Med 2021; 384:252-260