National Centre for Pharmacoeconomics

National Centre for

NCPE Ireland

Onasemnogene abeparvovec (Zolgensma®). HTA ID: 20021

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 for further information.