Onasemnogene abeparvovec (Zolgensma®) is indicated for the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
|NCPE Assessment Process||Complete|
|Rapid review commissioned||23/04/2020|
|Rapid review completed||13/05/2020|
|Rapid Review outcome||A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of onasemnogene abeparvovec (Zolgensma®) compared with the current standard of care.|
This HTA is being undertaken as part of the BENELUXA collaboration. It is a joint assessment between Ireland, Netherlands and Belgium with Austria acting as reviewer. Please see www.beneluxa.org for further information.