Advice

following a full submission considered under the ultra-orphan process:


inotersen (TegsediĀ®) 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 details

Medicine name:
inotersen (Tegsedi)
SMC ID:
SMC2188
Indication:

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
Full
Status
Accepted
Date advice published:
12 August 2019