following a full submission assessed under the ultra-orphan medicine process:
patisiran (Onpattro®) is accepted for use within NHSScotland.
Indication under review: the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
In a phase III study of adults with hATTR amyloidosis and polyneuropathy, patisiran was associated with significant improvements compared with placebo, measured by the change in modified neuropathy impairment score +7 (mNIS+7) from baseline to 18 months.
This SMC advice takes account of the benefit of a Patient Access Schemes (PAS) that improves the cost- effectiveness of patisiran. This advice is contingent upon the continuing availability of the PAS in NHSScotland or a list price that is equivalent or lower.
This advice takes account of views from a Patient and Clinician Engagement (PACE) meeting.
- Medicine name:
- patisiran (Onpattro)
- SMC ID:
For the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
- Pharmaceutical company
- Alnylam Pharmaceuticals
- BNF chapter
- Central nervous system
- Submission type
- Date advice published
- 10 June 2019