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NICE recommends inotersen for treating an ultra-rare form of amyloidosis

The National Institute for Health and Care Excellence (NICE) recommends inotersen (Tegsedi) for the treatment of hereditary transthyretin (TTR) amyloidosis in its latest 'highly specialised technologies' guidance.

Hereditary TTR amyloidosis is an ultra-rare inherited disorder resulting from mutations in the TTR gene. The mutation causes the liver to make abnormal TTR protein, which builds up as deposits inside body tissues (amyloidosis), consequently altering the structure and function of affected tissues.

Stage 1 involves mild sensory and motor neuropathy in the lower limbs, and stage 2 involves progressive neuropathy in the lower limbs along with the development of symptoms in the hands. Stage 3 involves severe sensory and motor neuropathy of all limbs making the patient bedridden. The condition severely affects the quality of life of patients, and their families and carers. Supportive care is the only current treatment available.

Inotersen is a novel molecule that inhibits the production of TTR protein. It is recommended for the treatment of stage 1 and stage 2 polyneuropathy in adults with hereditary TTR amyloidosis. The recommended dose for inotersen is 284 mg once weekly self-administered as a subcutaneous injection. It is available to the NHS at a discounted price under a commercial arrangement with the manufacturer.


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