Advice

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 details

Medicine name:
patisiran (Onpattro)
SMC ID:
SMC2157
Indication:

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
Full
Status
Accepted
Date advice published:
10 June 2019