following a full submission assessed under the orphan process:
risdiplam (Evrysdi®) is accepted for use within NHSScotland.
Indication under review: 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 [survival of motor neuron 2] copies.
Evidence from two phase II/III studies has indicated that risdiplam improves motor milestones and motor function in patients with type 1, 2 and 3 SMA.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
- Medicine name:
- risdiplam (Evrysdi)
- SMC ID:
For the treatment of 5q spinal muscular atrophy (SMA) in patients two 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.
- Pharmaceutical company
- BNF chapter
- Musculoskeletal and joint diseases
- Submission type
- Date advice published
- 07 February 2022