ATMPs in Germany - Casgevy®

👉 Casgevy is the first CRISPR/Cas9 gene-edited therapy.

Casgevy (active substance: exagamglogene autotemcel) was authorized in February 2024 for the treatment of sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). The ATMP Casgevy is the first authorized CRISPR/Cas9 gene-editing therapy. Pivotal trials were the single-arm studies “study 111” for TDT and “study 121” for SCD. Patients from both studies were invited to enroll in the long-term follow-up study “study 131”. Casgevy was not yet launched in Germany and thus, is not available.
 

Therefore, no🏆German HTA started and no 💵 price is listed.

💡 However, the German HTA body currently examines Casgevy to be subject to the ATMP Quality Assurance Guideline and routine practice data collection.