Browse Conditions Browse Medicines

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.

Sources
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

New medicines and vaccines that are under additional monitoring have an inverted black triangle symbol (▼) displayed in their package leaflet and summary of product characteristics, together with a short sentence explaining what the triangle means – it does not mean the medicine is unsafe. You should report all suspected adverse drug reactions (ADRs) for these products. ADRs can be reported by your doctor, pharmacist or online via the Yellow Card system.

Reporting of suspected adverse reactions

Reporting suspected adverse reactions (side effects) after authorisation of the medicinal product is important. It allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare professionals or patients are asked to report any suspected adverse reactions via the Yellow Card Scheme at yellowcard.mhra.gov.uk or search for MHRA Yellow Card in the Google Play or Apple App Store.


Disclaimer: This site is designed to offer information for general educational purposes only. The health information furnished on this site and the interactive responses are not intended to be professional advice and are not intended to replace personal consultation with a qualified physician, pharmacist, or other healthcare professional. We cannot provide individual medical advice. You must always seek the advice of a professional for questions related to a disease, disease symptoms, and appropriate therapeutic treatments.


About Transthyretin amyloidosis About Polyneuropathy About medicine