Onpattro

Pronunciation: on-pat-tro
Generic name: patisiran
Dosage form: solution for intravenous infusion
Drug class: Miscellaneous metabolic agents

Medically reviewed by Carmen Pope, BPharm. Last updated on May 7, 2024.

What is Onpattro?

Onpattro (patisiran) is an injectable transthyretin-directed small interfering RNA that may be used to treat polyneuropathy (multiple nerve damage) caused by hereditary transthyretin-mediated amyloidosis (ATTR) in adults. ATTR occurs when your liver produces faulty transthyretin (TTR) proteins and TTR deposits accumulate in organs and tissues, most commonly the peripheral nerves.

Onpattro (patisiran) works by breaking down mutant and wild-type transthyretin (TTR) proteins through RNA interference. TTR is an important protein primarily produced by the liver, that carries the thyroid hormone thyroxine and retinol (vitamin A) throughout the body. Onpattro decreases serum levels of TTR protein and protein deposits in tissues, relieving the symptoms of polyneuropathy.

Onpattro was FDA-approved on August 10, 2018.

Onpattro side effects

Onpattro can cause serious side effects, such as allergic reactions, infusion-related reactions, and vision problems. See warnings below.

The most common side effects of Onpattro occurring in at least 10% of people administered it include upper respiratory tract infections (with symptoms such as stuffy nose, sneezing, and sore throat) and infusion-related reactions.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to Alnylam Pharmaceuticals at 1-877-256-9526 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Warnings

Get emergency medical help if you have signs of an allergic reaction to Onpattro with symptoms such as hives; difficulty breathing; and swelling of your face, lips, tongue, or throat.

A reaction may occur while you are being administered Onpattro, in some people this could be serious or life-threatening. Tell your medical caregiver if you feel warm, dizzy, nauseated, light-headed, itchy, sweaty, or have a headache, chest tightness, back pain, trouble breathing, or swelling in your face.

Onpattro reduces serum vitamin A levels. Your healthcare provider will talk to you about taking the recommended daily allowance of vitamin A. Do not take higher than recommended doses of vitamin A to try to achieve normal serum vitamin A levels during treatment, as serum vitamin A levels do not reflect the total vitamin A in the body. Talk to your healthcare provider who should refer you to an ophthalmologist if you develop eye symptoms suggestive of vitamin A deficiency, such as night blindness.

Before receiving Onpattro

Before receiving Onpattro tell your doctor about all your medical conditions or allergies. Especially tell your doctor if you are:

Pregnant or intending to become pregnant
Breastfeeding.

Pregnancy

Onpattro may harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant. If you are pregnant, your name may be listed on a pregnancy registry to track the effects of Onpattro on the baby.

Breastfeeding

It may not be safe to breastfeed while using this medicine. Ask your doctor about any risks.

How is Onpattro administered?

Onpattro is given by a healthcare provider as a slow infusion into a vein over about 80 minutes.

It is usually given once every 3 weeks.

The recommended dosage of Onpattro is based on your weight.

Weight less than 100 kg: 0.3 mg/kg every 3 weeks.
Weight of 100 kg or more: 30 mg every 3 weeks.
- Your dosage may change if you gain or lose weight.

You may be given other medications, such as corticosteroids, acetaminophen, and antihistamines to help prevent serious side effects or an infusion reaction.

Tell your caregivers if you feel burning, pain, or swelling around the IV needle when Onpattro is injected.

Your doctor may have you take vitamin A while you are using Onpattro. Take only the amount of vitamin A your doctor has prescribed, especially if you are pregnant. An overdose of vitamin A can cause vision problems or other serious side effects.

Call your doctor at once if you have vision problems (especially at night) while you are taking Onpattro.

You may need frequent medical tests. Even if you have no symptoms, tests can help your doctor determine if this medicine is effective. You may also need frequent eye exams.

What happens if I miss a dose?

Call your doctor for instructions if you miss an appointment for your Onpattro.

What happens if I overdose?

Since Onpattro is given by a healthcare professional in a medical setting, an overdose is unlikely to occur.

What should I avoid while using Onpattro?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

What other drugs will affect Onpattro?

Other drugs interact with Onpattro, including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

No formal clinical drug interaction studies have been performed but Onpattro is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.

See the Onpattro package insert for more information about drug interactions.

Storage

Store in the refrigerator at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard the vial if it has been frozen.

If refrigeration is unavailable, Onpattro can be stored at room temperature up to 25°C (up to 77°F) for up to 14 days.

Once diluted, the solution should be administered immediately after preparation. If not used immediately, store in the infusion bag at room temperature (up to 30°C [86°F]) for up to 16 hours (including infusion time). Do not freeze.

Onpattro ingredients

Active: patisiran sodium

Inactive: MC-3, 1,2-dimyristoyl-SN-glycero-3-carboxaminopropylpolyethylene glycol 2000 methyl ether, 1,2-distearoyl-sn-glycero-3-phosphocholine, cholesterol, sodium phosphate, dibasic, heptahydrate, monobasic potassium phosphate, sodium chloride, water.

Available as a lipid complex injection: 10 mg/5 mL (2 mg/mL) in a single-dose vial.

Manufacturer

Alnylam Pharmaceuticals, Inc.

Popular FAQ

What type of drug is Onpattro?

Onpattro (patisiran) is the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatments. Onpattro 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. Continue reading