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Molecule

Patisiran

Also known as patisiran sodium
Available on requestOrphanCold chainNeurology
Presentations
1
Brands
1
Manufacturers
1
Available from stock
0

Patisiran is a small interfering RNA indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. It is available as lipid complex injection for intravenous use. MedsAcross supplies Patisiran for export from India to licensed hospitals, importers and physicians acting for named patients, with the documents each country requires.

Information from the US FDA prescribing information, retrieved 12 November 2026. Not yet medically reviewed.How we review

Key facts

Pharmacological class
Rna, Small Interfering RNA, Transthyretin-directed RNA Interaction, Decreased RNA Integrity, Increased Protein Breakdown
ATC classification
N07XX12 Patisiran · Other nervous system medicines, subgroup N07X, subgroup N07XX
US FDA first approval
10 August 2018
EU status
Authorised
Orphan designation
Yes
WHO essential medicine
Not recorded
India (CDSCO)
Not yet confirmed against CDSCO approval lists
Routes
Intravenous
Dosage forms
Lipid complex injection
Identifiers
UNII WO0YM16LKGRxCUI 2053490CAS 1420706-45-1DrugBank DB14582Q40441442

Clinical information

From the regulator-approved product information. Each section names its source.

Uses

ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. ONPATTRO contains a transthyretin-directed small interfering RNA and is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

Source: US FDA prescribing information

How it works

Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Source: US FDA prescribing information

Dosing overview

For patients weighing less than 100 kg, the recommended dosage is 0.3 mg/kg every 3 weeks by intravenous infusion. For patients weighing 100 kg or more, the recommended dosage is 30 mg. Premedicate with a corticosteroid, acetaminophen, and antihistamines. Filter and dilute prior to administration. Infuse over approximately 80 minutes

Source: US FDA prescribing information

Warnings and precautions

Infusion-related reactions: Monitor for signs and symptoms during infusion. Slow or interrupt the infusion if clinically indicated. Discontinue the infusion if a serious or life-threatening infusion-related reaction occurs. Reduced serum vitamin A levels and recommended supplementation: Supplement with the recommended daily allowance of vitamin A. Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur

Source: US FDA prescribing information

Side effects

The following clinically significant adverse reactions are described elsewhere in the labeling: Infusion-Related Reactions The most frequently reported adverse reactions (that occurred in at least 10% of ONPATTRO-treated patients and at least 3% more frequently than on placebo) were upper respiratory tract infections and infusion-related reactions

Source: US FDA prescribing information

Storage

Store at 2°C to 8°C (36°F to 46°F). Do not freeze. If refrigeration is not available, ONPATTRO can be stored at room temperature up to 25°C (up to 77°F) for up to 14 days. For storage conditions of ONPATTRO after dilution in the infusion bag, see Dosage and Administration.

Source: US FDA prescribing information

For healthcare professionals and buyers. This is not medical advice. Treatment decisions belong to the prescriber.

All presentations

1 presentations of Patisiran, sorted by availability.

MedicineManufacturerRegisterAvailability
Onpattro 10 mg/5 mL lipid complex injectionIntravenous Cold chainAlnylam PharmaceuticalsUS FDAAvailable on request

Brands by market

1 brand names across 1 markets.

Brand names by country

United States

Manufacturers

Regulatory status

Approval facts from US FDA, EMA and CDSCO data, and registration status by country where we hold register data.

RegisterStatusSource
US FDAFirst approved 10 August 2018Drugs@FDA
EU (EMA)AuthorisedEMA medicines data
India (CDSCO)Not yet confirmed against CDSCO approval lists—
AustriaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
BelgiumRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
BulgariaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
CroatiaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
CyprusRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
CzechiaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
DenmarkRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
EstoniaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
FinlandRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
FranceRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
GermanyRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
GreeceRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
HungaryRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
IcelandRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
IrelandRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
ItalyRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
LatviaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
LiechtensteinRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
LithuaniaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
LuxembourgRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
MaltaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
NetherlandsRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
NorwayRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
PolandRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
PortugalRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
RomaniaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
SlovakiaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
SloveniaRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
SpainRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
SwedenRegisteredEuropean Medicines Agency (central authorisation)Checked 12 November 2026
United StatesRegisteredUS FDA (NDC directory, openFDA)Checked 12 November 2026

Conditions

Conditions linked to Patisiran in public data. This is not a list of approved uses, see the clinical information.

Questions and answers

Is Patisiran available for export from India?

Yes, on request. MedsAcross lists 1 presentations of Patisiran and sources them for licensed hospitals, importers and physicians against valid import documents.

What brand names does Patisiran have?

The catalogue lists 1 brand names for Patisiran, including Onpattro.

Is Patisiran approved by the US FDA?

Yes. The US FDA first approved Patisiran on 10 August 2018, according to Drugs@FDA.

Is Patisiran authorised in the European Union?

Yes. Patisiran has a central EU marketing authorisation (status: authorised), according to European Medicines Agency data.

Does Patisiran need cold chain shipping?

At least one listed presentation of Patisiran must be kept cold, so it is shipped in temperature-controlled packaging. Check the storage line on each presentation.

Which ATC class is Patisiran in?

Patisiran is classified under ATC code N07XX12 (Patisiran).

References

  1. Onpattro prescribing information (Alnylam Pharmaceuticals). U.S. National Library of Medicine, DailyMed. Retrieved 8 September 2025
  2. Onpattro: European public assessment report (EPAR). European Medicines Agency. Retrieved 21 September 2026

Request Patisiran for export supply

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