NICE issue initial negative decisions on inotersen and patisiran

The UK’s National Institute for Health and Care Excellence (NICE) has published Evaluation Consultation Documents on its assessments of inotersen and patisiran.

In its draft guidance, NICE recognises that both drugs reduce disability, increase quality of life and are innovative. However, there remain some questions about the cost-effectiveness of both drugs, therefore neither can be recommended for treating hereditary transthyretin-related amyloidosis at this time. However, it is important to keep in mind that these are initial decisions and further discussions regarding the cost effectiveness of the drugs will be held. ARC expects that, NICE, the manufacturers and NHS England will continue to work towards making both drugs available to patients in England and Wales. Final decisions will be made early in the new year.

Inotersen (Tegsedi®) is a novel, antisense oligonucleotide that inhibits production of transthyretin (TTR) in adults with hereditary transthyretin-related (hTTR) amyloidosis. Inotersen has a marketing authorisationfor ‘the treatment of stage 1 or stage 2 polyneuropathy in adults with hATTR amyloidosis’.

Patisiran (Onpattro®) is a ribonucleic acid interference agent that suppresses transthyretin (TTR) production by the liver (including abnormal TTR). It is administered once every 3 weeks by intravenous infusion at a dose of 0.3 mg/kg. It has a marketing authorisation in the EU for treating ‘hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy’.

For further information on the NICE appraisals: click here >>

Information on inotersen >> 

Information on patisiran >>


320 Nevada Street, Suite 210 Newton, MA 02460 USA | 617-467-5170

ARC is a 501(c)(3) nonprofit organization