Inotersen for treating hereditary transthyretin amyloidosis

Inotersen for treating hereditary transthyretin amyloidosis (Highly specialised technologies guidance [HST9])

Evidence-based recommendations on inotersen (Tegsedi) for stage 1 and stage 2 polyneuropathy in adults with hereditary transthyretin amyloidosis.

Hereditary transthyretin (hATTR) amyloidosis is an ultra-rare condition caused by inherited mutations in the transthyretin (TTR) gene. This causes the liver to produce abnormal TTR protein, which accumulates as deposits in body tissues (amyloidosis). These deposits can disrupt the structure and damage the function of affected tissues.

Because hATTR amyloidosis can affect tissues throughout the body, people may have a range of symptoms affecting 1 or more systems. These can include the autonomic nervous system, peripheral nerves, heart, gastrointestinal system, eyes and central nervous system.

Inotersen (Tegsedi, Akcea Therapeutics) is a novel, first-in-class 2'-O-2-methoxyethyl phosphorothioate antisense oligonucleotide that inhibits production of transthyretin (TTR) in adults with hereditary transthyretin (hATTR) amyloidosis.

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