Risdiplam (Evrysdi™) is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
|NCPE Assessment Process||Complete|
|Rapid review commissioned||15/03/2021|
|Rapid review completed||29/03/2021|
|Rapid Review outcome||A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of risdiplam compared with the current standard of care.|