following a full submission considered under the ultra-orphan process:
inotersen (Tegsedi®) is accepted for use within NHSScotland.
Indication under review: for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
In a phase II/III study of adults with hATTR and polyneuropathy, inotersen was associated with significantly less worsening compared with placebo, measured by the change in modified neuropathy impairment score +7 (mNIS+7) and in Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire from baseline to 66 weeks.
This SMC advice takes account of the benefits of a Patient Access Scheme (PAS) that improves the cost-effectiveness of inotersen. 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 the views from a Patient and Clinician Engagement (PACE) meeting.
- Medicine name:
- inotersen (Tegsedi)
- SMC ID:
For the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
- Pharmaceutical company
- Akcea Therapeutics UK Ltd
- BNF chapter
- Central nervous system
- Submission type
- Date advice published:
- 12 August 2019