The National Institute for Health and Care Excellence (NICE) recommends patisiran (Onpattro) for the treatment of hereditary transthyretin (TTR) amyloidosis in its latest 'highly specialised technologies' guidance.
Patisiran is recommended for the treatment of hereditary TTR amyloidosis in adults with stage I or II polyneuropathy. 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.
Patisiran is a ribonucleic acid interference agent which can suppress TTR. The recommended dose of patisiran is 0.3 mg/kg to be administered intravenously once every three weeks. Most common adverse reactions reported during treatment include peripheral oedema, infusion-related reactions, infections, vertigo, dyspnoea, dyspepsia, erythema, arthralgia and muscle spasms.
Evidence has shown patisiran to reduce disability and improve quality of life in patients with hereditary TTR amyloidosis. Evidence also suggests that it may arrest the progression of amyloidosis and potentially reverse it in the long term. The drug should be used only when provided by the company in agreement with the commercial arrangement.