Onpattro
What Onpattro is used for
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
Key facts
- Brand
- Onpattro
- Active substance
- patisiran
- Disease area
- Amyloidosis, Familial
- Medicine group
- Other nervous system drugs
- ATC code
- N07
- Marketing authorisation holder
- Alnylam Netherlands B.V.
- Authorised in the EU since
- 2018
Official information
In the USA
Same active substance: Onpattro
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Onpattro used for?
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
What is the active substance in Onpattro?
Onpattro contains patisiran.
Who makes Onpattro?
The EU marketing authorisation holder is Alnylam Netherlands B.V..
Is Onpattro an orphan medicine?
Yes. Onpattro is designated an orphan medicine in the EU, for a rare disease (Amyloidosis, Familial).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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