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Tegsedi▼ gets approval for use in Scotland

Tegsedi▼, for Transthyretin amyloidosis from Akcea Therapeutics UK Ltd

The Scottish Medicines Consortium (SMC) have approved Akcea Therapeutic's medicine inotersen, brand name Tegsedi▼, for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).

This means that Tegsedi will be available as an option for patients being treated under the NHS in Scotland.

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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.

In patients with hATTR, a protein called transthyretin which circulates in the blood is defective and breaks easily. The broken protein forms amyloid deposits in tissues and organs around the body, including around nerves, where it interferes with their normal functions.

The active substance in Tegsedi, inotersen, is an ‘antisense oligonucleotide’, a very short piece of synthetic genetic material that has been designed to attach to and block the genetic material of the cell responsible for producing transthyretin. This reduces production of transthyretin, thereby reducing the formation of amyloids and relieving the symptoms of hATTR amyloidosis.

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

European Medicines Agency website page for Tegsedi
Accessed 13/08/19
Links available in External Resources

SMC information on Tegsedi

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Reporting of suspected adverse reactions

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