Buy Onpattro (patisiran) patisiran

Onpattro (patisiran) is an FDA-approved orphan drug developed by Alnylam Pharmaceuticals for treating hereditary ATTR amyloidosis with polyneuropathy. It represents a breakthrough in neurology as the first RNAi therapeutic targeting transthyretin (TTR) production. The drug was granted orphan drug designation due to the rare nature of hereditary ATTR amyloidosis.

Strength

2 mg/mL

Pack Size

1 VIAL, SINGLE-DOSE in 1 CARTON (71336-1000-1) / 5 mL in 1 VIAL, SINGLE-DOSE

Manufacturer / TM Owner

Alnylam Pharmaceuticals

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Uses / Indications

Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in patients with polyneuropathy. This rare genetic disorder causes mutant transthyretin protein to misfold and accumulate in peripheral nerves, leading to progressive neurological dysfunction. Onpattro works through RNA interference (RNAi) technology, silencing the TTR gene and reducing transthyretin protein production in the liver by approximately 80%. This mechanism halts disease progression and can improve neuropathic symptoms. The drug is administered as an intravenous infusion every three weeks. Patients typically include adults with stage 1 or stage 2 polyneuropathy, though it may benefit earlier-stage patients. The drug addresses an unmet medical need in this orphan population, as hereditary ATTR amyloidosis previously had limited disease-modifying treatment options.

Side Effects

Common side effects of Onpattro include infusion-related reactions such as flushing, back pain, and chest discomfort, which typically occur during or shortly after administration. Other reported adverse events include arthralgia, dyspnea, and transient liver enzyme elevations. Serious adverse events are rare but may include severe infusion reactions and hepatotoxicity. Patients require regular monitoring including liver function tests, platelet counts, and renal function before each infusion. Premedication with corticosteroids and antihistamines is recommended to minimize infusion-related reactions. Vitamin A supplementation is required as TTR reduction may lower vitamin A transport.

Precautions & Warnings

Onpattro is contraindicated in patients with known hypersensitivity to patisiran or any excipients. Caution is warranted in patients with severe hepatic impairment or thrombocytopenia. Pregnancy category is not established; women of childbearing potential should use effective contraception. Limited pediatric data exists; use in children requires careful consideration. Patients with renal impairment may require dose adjustments or additional monitoring. Those with cardiac amyloidosis should be monitored closely. Vitamin A supplementation is mandatory to prevent deficiency. Patients should avoid live vaccines during treatment. Infusion reactions require appropriate management protocols and medical supervision.

Avoid Interactions

No major drug-drug interactions have been formally documented with Onpattro. However, caution is advised when combining with other hepatotoxic agents due to potential liver enzyme elevation. Vitamin A supplementation is required and should not exceed recommended doses to avoid toxicity. NSAIDs and anticoagulants may require monitoring in patients with thrombocytopenia. Patients should inform healthcare providers of all medications, supplements, and herbal products. Alcohol consumption should be minimized given hepatic considerations.

Frequently Asked Questions

Onpattro is used to treat hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy in adults. It works by reducing the production of transthyretin protein in the liver, slowing disease progression and improving neuropathic symptoms. This orphan drug addresses a rare genetic disorder that previously had limited treatment options.

Onpattro is administered as an intravenous infusion every three weeks. Each infusion typically lasts 80 minutes and is given in a healthcare setting. Patients receive premedication with corticosteroids and antihistamines to reduce infusion-related reactions.

Common side effects include infusion-related reactions such as flushing, back pain, and chest discomfort. Other side effects may include joint pain, shortness of breath, and elevated liver enzymes. Most infusion reactions are mild to moderate and manageable with premedication.

Onpattro is manufactured by Alnylam Pharmaceuticals, a biopharmaceutical company specializing in RNA interference therapeutics.

Yes, Onpattro received orphan drug designation from the FDA due to the rare nature of hereditary ATTR amyloidosis. This designation provides regulatory incentives and support for developing treatments for rare diseases affecting small patient populations.

Published by GNH India Pharmaceuticals Limited

Last updated: 06 Jun 2026

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