patisiran
Onpattro · ATC code N07
- Key facts
- Active substance
- patisiran
- ATC code
- N07
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Onpattro 2 mg/mL concentrate for solution for infusion
- Marketing authorisation holder
- Alnylam Netherlands B.V.
- Information as of
- 2026-10-28
Key facts
Patisiran (Onpattro) is a small interfering RNA (siRNA) that reduces transthyretin production. It is given by infusion for hereditary transthyretin amyloidosis with polyneuropathy.
What it is used for
- Hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy.
Dosing
- 300 micrograms per kg body weight by intravenous infusion once every 3 weeks, based on actual body weight; for patients ≥ 100 kg the maximum is 30 mg.
- Premedication at least 60 minutes before each infusion: IV dexamethasone 10 mg (or equivalent), oral paracetamol 500 mg, an IV H1 blocker (e.g. diphenhydramine 50 mg) and an IV H2 blocker (e.g. famotidine 20 mg).
- Vitamin A supplementation of approximately 2 500 IU per day.
- Give a missed dose as soon as possible; for the subsequent schedule see the full text.
Contraindications
- Severe hypersensitivity (e.g. anaphylaxis) to the active substance or any of the excipients.
Important warnings
- Infusion-related reactions (e.g. flushing, back pain, nausea, dyspnoea, hypotension, syncope), mostly within the first 2 infusions; discontinue for a serious reaction.
- Reduces vitamin A levels; correct deficiency before starting and refer for eye assessment with symptoms such as night blindness.
- Continuing after progression to stage 3 polyneuropathy is a physician's decision.
Side effects
- Very common: peripheral oedema (29.7%) and infusion-related reactions (18.9%).
- Common: bronchitis, sinusitis, rhinitis, vertigo, dyspnoea, dyspepsia, erythema, arthralgia, muscle spasms.
- Uncommon: extravasation.
Interactions
- No formal interaction studies; no cytochrome P450 interactions expected; net effect on CYP2B6 substrates (e.g. bupropion, efavirenz) in vivo is unknown.
Pregnancy and breastfeeding
- Both too high and too low vitamin A levels may increase the risk of foetal malformation; exclude pregnancy before starting and use effective contraception.
- If planning pregnancy, stop Onpattro and vitamin A supplementation and wait for vitamin A levels to normalise.
Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
Common questions – ask Medelix
For patients
Package leaflet, verbatim
This product has no separate US patient information.
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- OnpattroAlnylam Netherlands B.V. · EMEA/H/C/004699
Source: European Medicines Agency (EMA), product information
Brand names in 3 more European countries
- Iceland
- Onpattro
- Liechtenstein
- Onpattro
- Norway
- Onpattro
EMA – Article 57 product data (EU/EEA), 2026-10-29
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Another question about this substance?
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Sources
- European Medicines Agency. Onpattro – EPAR, Product Information. 2026-10-28. https://www.ema.europa.eu/en/documents/product-information/onpattro-epar-product-information_en.pdf
Editorial: Medelix editorial team · last updated 2026-10-28 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.