This medicine is authorised for use in the European Union.


Onpattro is a medicine 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.

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.

Onpattro contains the active substance patisiran.

This EPAR was last updated on 02/06/2023

Authorisation details

Product details
Agency product number
Active substance
patisiran sodium
International non-proprietary name (INN) or common name
Therapeutic area (MeSH)
Amyloidosis, Familial
Anatomical therapeutic chemical (ATC) code
Accelerated assessmentAccelerated assessment

This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
Alnylam Netherlands B.V.
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Antonio Vivaldistraat 150
1083 HP Amsterdam

Product information

26/05/2023 Onpattro - EMEA/H/C/004699 - PSUSA/00010715/202208

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Product information documents contain:

You can find product information documents for centrally authorised human medicines on this website. For centrally authorised veterinary medicines authorised or updated from February 2022, see the Veterinary Medicines Information website.

Pharmacotherapeutic group

Other nervous system drugs

Therapeutic indication

Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.

Assessment history

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