PTJA17 – Elivaldogene autotemcel (eli-cel) for the treatment of cerebral adrenoleukodystryophy (CALD) – Final assessment now available
This is the pharmaceutical Joint Assessment PTJA17 – Elivaldogene autotemcel (eli-cel) for the treatment of cerebral adrenoleukodystrophy (CALD). In July, 2021, the European Commission granted marketing authorisation for Skysona® (eli-cel) for the treatment of cerebral adrenoleukodystrophy. This Joint Assessment aims to compare the clinical effectiveness and safety of eli-cel in the target patient populations with relevant comparators according to the national requirements of EUnetHTA partners.
Below is the documentation provided by the Joint Assessment authoring team (project plan, assessment report and responses to the factual accuracy check performed by bluebird bio B.V.; and the Core Submission Dossier prepared by bluebird bio B.V., the Marketing Authorisation Holder of eli-cel.
A revised version of the assessment report originally posted on 16/08/2021 was published on 09/09/2021. An error was identified on 09/09/2021 regarding the reported numbers of patients that discontinued their treatment due to adverse events in table 0.2. and table 4.13, and regarding the reported outcome numbers of serious adverse events related to therapy in table 4.12. For further details, please see the version history table in the assessment report.
For any questions regarding the assessment, please contact WP4_Pharmaceuticals@zinl.nl
Below is the documentation provided by the Joint Assessment Authoring Team.