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.
PTJA17 – Final Project Plan
PTJA17 – Final Assessment Report
PTJA17 – MAH Core Submission Dossier
PTJA17 – External Comments – Factual Accuracy Check MAH
This website was produced under the Third EU Health Programme through a service contract with the European Health and Digital Executive Agency (HaDEA) acting under the mandate from the European Commission. The information and views set out in this website are those of the author(s) and do not necessarily reflect the official opinion of the Commission/Executive Agency. The Commission/Executive Agency do not guarantee the accuracy of the data included in this website. Neither the Commission/Executive Agency nor any person acting on the Commission’s/Executive Agency’s behalf may be held responsible for the use which may be made of the information contained therein.
©2021 EUnetHTA All rights reserved.