GNH USA

Europe — EMA

Onpattro (Patisiran)

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

Patisiran

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What is Patisiran?

Patisiran, the active ingredient in Onpattro, is a small interfering RNA (siRNA) therapeutic presented as a lipid nanoparticle infusion for intravenous use. It treats hereditary transthyretin‑mediated amyloidosis in adult patients, addressing both polyneuropathy and cardiomyopathy manifestations.

GNH USA supplies Onpattro as a licensed, GDP‑compliant international pharmaceutical supplier serving hospitals worldwide. The company provides regulated sourcing, quality assurance and logistics support to meet global demand.

What is Patisiran used for?

Onpattro (patisiran) is indicated for hereditary transthyretin‑mediated amyloidosis, a rare protein‑misfolding disorder affecting the nervous and cardiac systems.

- Hereditary transthyretin amyloidosis (polyneuropathy): slows disease progression and improves neurological function. - Hereditary transthyretin amyloidosis (cardiomyopathy): stabilizes cardiac function and reduces deterioration. - Familial amyloid polyneuropathy: alleviates sensory loss and motor impairment.

What are the side effects of Patisiran?

Common Side Effects: - Infusion‑related reactions: flushing, chills, or rash. - Nausea and vomiting. - Fatigue or weakness. - Peripheral edema. - Headache. - Dizziness.

Serious or Rare Side Effects: - Anaphylaxis or severe hypersensitivity. - Significant elevation of hepatic enzymes. - Thrombocytopenia. - Acute renal impairment. - Cardiac arrhythmias. - Severe infusion‑related cytokine release.

What precautions apply to Patisiran?

Before initiating therapy, evaluate baseline organ function and monitor throughout treatment.

- Renal function: assess serum creatinine and eGFR before each infusion. - Hepatic function: monitor ALT, AST, and bilirubin levels regularly. - Cardiovascular status: evaluate for heart failure signs prior to dosing. - Pregnancy and lactation: avoid use unless benefits clearly outweigh potential risks. - Infusion reactions: administer under qualified medical supervision with emergency equipment available. - Store at 2-8°C: keep refrigerated, protect from light, do not freeze or shake.

What does Patisiran interact with?

Patisiran may interact with other agents that affect immune function or hepatic metabolism.

Major Interactions (Avoid): - Concurrent siRNA therapies: risk of additive RNAi effects. - Live vaccines: may compromise vaccine efficacy. - Immunosuppressants: increased infection susceptibility. - Other lipid nanoparticle formulations: potential overlapping toxicity. - Strong CYP inducers: may alter metabolism of co‑administered drugs.

Moderate Interactions (Monitor Closely): - Warfarin: monitor INR as hepatic changes can affect anticoagulation. - Statins: watch for muscle toxicity. - Antihypertensives: observe blood pressure for fluid‑shift related changes. - Diuretics: adjust dosing if significant edema occurs. - Antiplatelet agents: monitor for bleeding tendencies.

Frequently asked questions

What condition is Onpattro used to treat?+
Onpattro (patisiran) is prescribed for hereditary transthyretin‑mediated amyloidosis, a rare disorder that can cause progressive nerve damage (polyneuropathy) and heart involvement (cardiomyopathy) in adults.
How does patisiran work in the body?+
Patisiran is a small interfering RNA that binds to transthyretin messenger RNA in liver cells, leading to its degradation. This reduces production of both mutant and normal transthyretin protein, lowering the amount of amyloid‑forming protein that deposits in tissues.
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. Specific dosage instructions are provided in the product’s prescribing information.
Is Onpattro safe during pregnancy or breastfeeding?+
Onpattro is not recommended for use during pregnancy or while breastfeeding unless the potential benefit justifies the potential risk to the fetus or infant. Women of childbearing potential should use effective contraception during treatment.
How do I order Onpattro from GNH USA?+
To request Onpattro, submit an enquiry on the product page at gnh-usa.com with 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 if needed) and import documentation.
How does Onpattro compare with other treatments for hATTR amyloidosis?+
Onpattro is an RNA‑interference therapy that reduces transthyretin production, whereas alternatives such as tafamidis stabilize the transthyretin protein and diflunisal act as off‑label stabilizers. The choice depends on disease phenotype, patient tolerance, and physician assessment of efficacy and safety.
What are the storage and handling requirements for Onpattro?+
Onpattro must be stored refrigerated at 2‑8 °C, protected from light, and must not be frozen or shaken. Prior to infusion, the product is prepared according to the manufacturer’s instructions in a controlled environment.
How is Onpattro administered to patients?+
Onpattro is given as an intravenous infusion, typically administered once every three weeks. The infusion is performed in a clinical setting by qualified healthcare professionals, with monitoring for infusion‑related reactions.
What are the most serious risks associated with Onpattro?+
Serious risks include severe hypersensitivity reactions (including anaphylaxis), significant liver enzyme elevations, thrombocytopenia, renal impairment, and cardiac arrhythmias. Patients should be closely monitored for these events throughout therapy.
Who should not use Onpattro?+
Onpattro is contraindicated in patients with known hypersensitivity to patisiran or any component of the formulation. It should also be avoided in individuals with uncontrolled severe cardiac disease or active infections without appropriate medical supervision.
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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