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NICE approve use of hereditary transthyretin amyloidosis (hATTR) treatment Tegsedi▼

Tegsedi▼, for Transthyretin amyloidosis and Polyneuropathy from Akcea Therapeutics UK Ltd

The National Institute for Health and Care Excellence (NICE) approved Akcea's inotersen, brand name Tegsedi for use in the NHS in England and Wales.

Tegsedi is used to treat polyneuropathy in patients with hereditary transthyretin amyloidosis (hATTR).

Polyneuropathy is damage or disease affecting peripheral nerves (peripheral neuropathy) in roughly the same areas on both sides of the body, featuring weakness, numbness, and burning pain.

NICE state that Tegsedi is recommended as an option for treating stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin-related amyloidosis.

Why NICE made these recommendations
Hereditary transthyretin-related amyloidosis is a rare condition that severely affects the quality of life of people with the condition, and their families and carers. Current treatment is supportive care.

Clinical trial evidence shows that inotersen slows progression of the disease considerably, although its long-term benefits are uncertain. Some assumptions in the economic modelling are also uncertain, particularly around the utility values and the healthcare costs. Despite the uncertainties, inotersen is likely to provide important clinical benefits for
people with hereditary transthyretin-related amyloidosis and value for money within the context of a highly specialised service. It is therefore recommended for use in the NHS.

About Tegsedi (source EMA)
Tegsedi is a medicine used to treat nerve damage caused by hereditary transthyretin amyloidosis (hATTR), a disease in which proteins called amyloids build up in tissues around the body including around the nerves.

Tegsedi is used in adult patients in the first two stages of the nerve damage (stage 1, when the patient is able to walk unaided, and stage 2, when the patient can still walk but needs help).

hATTR is rare, and Tegsedi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 26 March 2014.

Tegsedi was first made available in the EU in 2018. It is manufactured by Akcea Therapeutics.

European Medicines Agency website page for Tegsedi
Accessed 24/04/19
Links available in full article.

© NICE [2019] Inotersen for treating hereditary transthyretin-related amyloidosis [ID1242] Available from: See Link below. All rights reserved. Subject to Notice of rights
NICE guidance is prepared for the National Health Service in England. All NICE guidance is subject to regular review and may be updated or withdrawn. NICE accepts no responsibility for the use of its content in this product/publication.

The information provided by NICE was accurate at the time this article was issued.

NICE information on Tegsedi

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