GNH USA

Europe — EMA

Onpattro (Patisiran)

GNH USA supplies Onpattro (Patisiran), a RNAi therapeutic for hereditary transthyretin amyloidosis with polyneuropathy. Request a quote for global supply.

Patisiran

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What is Patisiran?

Patisiran, the active ingredient in Onpattro, is an RNA interference (RNAi) therapeutic presented as a lipid nanoparticle intravenous solution for infusion use. It is indicated for the treatment of hereditary transthyretin‑mediated amyloidosis in adults, primarily to address polyneuropathy and related disease manifestations.

GNH USA supplies Onpattro as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals, transplant centres and specialty clinics worldwide. Our global distribution network ensures timely delivery and compliance with local regulatory requirements. We provide full cold‑chain logistics and product support to meet the needs of advanced care facilities.

What is Patisiran used for?

Onpattro acts on the transthyretin (TTR) protein synthesis pathway to treat hereditary transthyretin amyloidosis.

Hereditary transthyretin amyloidosis (polyneuropathy): reduces hepatic TTR production, slowing progression of peripheral nerve damage. Hereditary transthyretin amyloidosis (cardiomyopathy): lowers circulating TTR levels, helping to stabilize cardiac function. Familial amyloid polyneuropathy (early‑stage): delays onset of functional impairment.

What are the side effects of Patisiran?

Common and serious adverse reactions have been reported with Onpattro therapy.

Common Side Effects: - Infusion‑related reactions (flushing, chills, back pain) - Nausea - Fatigue - Peripheral edema

Serious or Rare Side Effects: - Severe hypersensitivity or anaphylaxis - Significant elevation of liver enzymes (ALT/AST) - Thrombocytopenia or bleeding disorders

What precautions apply to Patisiran?

When using Onpattro, several safety considerations should be observed.

- Monitor liver function: check ALT/AST before each infusion and periodically during treatment. - Assess cardiac status: evaluate baseline ECG and echocardiography in patients with cardiomyopathy. - Observe for infusion reactions: have emergency medications ready during administration. - Use caution in pregnancy: limited data, weigh potential benefits against risks. - Avoid in patients with known hypersensitivity to siRNA or lipid components. - Store at 2-8°C: keep refrigerated, protect from light, do not freeze or shake.

What does Patisiran interact with?

Onpattro may interact with other medicines; some combinations require avoidance while others need close monitoring.

Major Interactions (Avoid): - Concomitant use of other hepatotoxic agents: increased risk of liver injury. - Co‑administration with strong CYP inducers: may alter lipid nanoparticle metabolism.

Moderate Interactions (Monitor Closely): - Anticoagulants or antiplatelet drugs: monitor for bleeding if thrombocytopenia occurs. - Live vaccines: assess immune response due to RNAi therapy. - Herbal supplements affecting liver enzymes: monitor hepatic parameters. - Other RNAi or gene‑silencing therapies: potential additive immune effects.

Frequently asked questions

What is Onpattro used for?+
Onpattro (patisiran) is indicated for the treatment of hereditary transthyretin‑mediated amyloidosis in adults, primarily to manage polyneuropathy and, in some regions, associated cardiomyopathy. It works by reducing the production of both mutant and wild‑type transthyretin protein, thereby slowing disease progression.
How does patisiran work?+
Patisiran is a small interfering RNA (siRNA) delivered in a lipid nanoparticle. After intravenous infusion, it enters liver cells and joins the RNA‑induced silencing complex, where it binds to transthyretin messenger RNA. This binding leads to degradation of the mRNA, preventing translation and markedly lowering circulating transthyretin levels.
What is the usual dose of Onpattro?+
The dosing regimen for Onpattro is determined by the prescribing physician based on the patient’s weight, disease severity, and clinical response. The prescriber sets the exact dose and infusion schedule, which is typically administered every three weeks under medical supervision.
Is Onpattro safe during pregnancy or breastfeeding?+
Data on the use of Onpattro in pregnancy and lactation are limited. It should only be used if the potential benefit justifies any potential risk to the fetus or infant. Women of child‑bearing potential should use effective contraception during treatment and for a period after the last dose.
How do I order Onpattro from GNH USA?+
To request Onpattro, submit an enquiry on the product page at gnh-usa.com, specifying the required quantity. GNH USA supplies hospitals, pharmacies and procurement teams worldwide, verifies trade or institutional credentials, and then provides pricing, availability, shipping details (including cold‑chain logistics if needed) and the necessary import documentation.
How does Onpattro compare with other treatments for hereditary transthyretin amyloidosis?+
Onpattro is an RNAi‑based therapy that reduces transthyretin production at the genetic level, whereas other options such as tafamidis stabilize the TTR protein and diflunisal act as kinetic stabilizers. The choice depends on disease stage, organ involvement, patient tolerance, and physician assessment of efficacy and safety profiles.
What are the storage and handling requirements for Onpattro?+
Onpattro must be stored refrigerated at 2‑8 °C. It should be kept in its original packaging, protected from light, and must not be frozen or shaken. Prior to infusion, the solution is allowed to reach room temperature as directed by the product label.
How is Onpattro administered?+
Onpattro is given as an intravenous infusion, typically over about 80 minutes, once every three weeks. The infusion is performed in a clinical setting where healthcare professionals can monitor the patient for any infusion‑related reactions and manage them promptly.
What are the most serious risks associated with Onpattro?+
Serious risks include severe hypersensitivity reactions (including anaphylaxis), significant elevations in liver enzymes indicating hepatic injury, and thrombocytopenia that can lead to bleeding complications. Patients should be monitored closely for these events throughout treatment.
Who should not use Onpattro?+
Onpattro is contraindicated in individuals with known hypersensitivity to patisiran, its lipid nanoparticle components, or any excipients in the formulation. Caution is also advised for patients with severe hepatic impairment, uncontrolled cardiac disease, or those who are pregnant without a clear benefit‑risk justification.
Information provided on this page is for educational use by procurement and clinical professionals. Always consult a licensed healthcare provider for medical advice and prescribing decisions. GNH USA supplies this product subject to applicable regulatory and licensing requirements.
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Product details

Drug
Patisiran
Route
Intravenous Use
Manufacturer
Alnylam Netherlands B.V.
Origin
EU

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