Extended indication - Sickle cell disease (SCD) - Beta-thalassemia, transfusion-dependent (TDT)
Therapeutic value No estimate possible yet
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)
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 comments Substance a.k.a. CTX001

Registration

Registration route Centralised (EMA)
ATMP Yes
Submission date 2022
Expected Registration 2023
Orphan drug Yes
Registration phase Clinical trials

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
Duration of treatment one-off
References NCT03655678
Additional comments Administered by IV infusion following myeloablative conditioning with busulfan.

Expected patient volume per year

References hematologienederland.nl
Additional comments 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
References https://www.fool.com/investing/2021/07/23/3-things-about-crispr-therapeutics-smart-investors/
Additional comments 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

There is currently nothing known about the possible total cost.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension No
References adisinsight; fabrikant

Other information

Additional comments Frangoul et al 2021. N Engl J Med 2021; 384:252-260