The National Institute for Health and Care Excellence (NICE) have recommended Alnylam's medicine patisiran, brand name Onpattro▼ as an option for treating hereditary transthyretin amyloidosis in adults with stage 1 and stage 2 polyneuropathy.
Onpattro is used to treat nerve damage caused by hereditary transthyretin (hATTR) amyloidosis, a disease in which abnormal proteins called amyloids build up in tissues around the body including around the nerves.
This decision means that Onpattro is available as a treatment option for patients in England and Wales.
You can follow developments with Onpattro by using our Medicines Tracker service which provides users with updates about the medicines they are interested in.
Why NICE made these recommendations
Hereditary transthyretin amyloidosis is a rare condition that severely affects the health and quality of life of people with the condition, as well as the quality of life of their families and carers. At the time of the evaluation, there were no disease-modifying treatments in widespread use.
Clinical trial evidence shows that patisiran reduces disability and improves quality of life, by enabling patients to return to work, carry out daily activities, participate in a more active family and social life, and maintain their independence and dignity. There is also evidence suggesting that patisiran may provide long-term benefits by stopping the progression of amyloidosis and potentially reversing it.
About Onpattro (source EMA)
Onpattro 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 amyloidosis is rare, and Onpattro was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 15 April 2011.
In patients with hATTR amyloidosis, 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 Onpattro, patisiran, is a ‘small interfering RNA’ (siRNA), 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 defective transthyretin, thereby reducing the formation of amyloids and relieving the symptoms of hATTR amyloidosis.
Onpattro was first made available in the EU in 2018. It is manufactured by Alnylam.
European Medicines Agency website page for Onpattro
Links available in External Resources
© NICE 
Patisiran for treating hereditary transthyretin amyloidosis. Highly specialised technologies guidance [HST10]. Published date: 14 August 2019
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 Onpattro
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