What type of drug is Onpattro?

Onpattro (patisiran) is called a small interfering ribonucleic acid (siRNA) treatment. It is used for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. Onpattro works by encasing siRNA in lipids and stops the production of disease-causing amyloid proteins.

Polyneuropathy of hereditary transthyretin-mediated amyloidosis is a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in nerves, the heart and other organs.

How is Onpattro given?

Onpattro is administered by intravenous (IV) infusion over approximately 80 minutes, every 3 weeks by a healthcare provider. Doses are based on your weight. You will probably receive this medicine at an infusion center, but may be eligible to receive it at home. Ask your doctor about this option.

At least one hour before your Onpattro infusion, you will be pre-medicated with a corticosteroid, acetaminophen, and antihistamines to help reduce possible infusion reactions. These reactions most commonly include flushing, back pain, nausea, stomach pain, shortness of breath, and headache. Side effects due to infusion reactions tend to lessen over time.

Are side effects common with Onpattro?

Upper respiratory tract infections and infusion-related reactions are the most common side effects with Onpattro.

Common side effects in studies with Onpattro that occurred in at least 5% of patients (5 out of every 100 patients) included:

• upper respiratory tract infections - (29% Onpattro vs. 21% placebo)
• infusion-related reactions - (19% Onpattro vs. 9% placebo)
• heartburn (dyspepsia) - (8% Onpattro vs. 4% placebo)
• shortness of breath (dyspnea) - (8% Onpattro vs. 0% placebo)
• muscle spasms - (8% Onpattro vs. 1% placebo)
• joint pain (arthralgia) - (7% Onpattro vs. 0% placebo)
• redness, flushing (erythema) - (7% Onpattro vs. 3% placebo)
• bronchitis - (7% Onpattro vs. 3% placebo)
• dizzy spells, feeling off-balance (vertigo) - (5% Onpattro vs. 1% placebo)

Related: More serious side information (in detail)

This is not all the information you need to know about Onpattro (patisiran) for safe and effective use. Review the full Onpattro information here, and discuss this information and any questions you have with your doctor or other health care provider.