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Patisiran

Generic Name: patisiran (PAT i SIR an)
Brand Name: Onpattro

Medically reviewed by Drugs.com on Aug 22, 2018 – Written by Cerner Multum

What is patisiran?

Patisiran works by decreasing a protein called transthyretin (TTR, made primarily in the liver). Hereditary transthyretin- mediated amyloidosis (hATTR) is a rare condition in which abnormal deposits of TTR protein build up in many parts of the body, interfering with normal function.

Patisiran is used to treat polyneuropathy (damage of multiple nerves throughout the body) in adults with hATTR. patisiran can help reduce symptoms such as pain, numbness, tingling, abnormal heartbeats, diarrhea, constipation, weakness, and problems with movement in your arms or legs.

Patisiran may also be used for purposes not listed in this medication guide.

Important Information

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You should not use patisiran if you are allergic to it.

Before using patisiran tell your doctor about all your medical conditions or allergies.

It is not known whether patisiran will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

It may not be safe to breast-feed while using this medicine. Ask your doctor about any risk.

How is patisiran given?

Patisiran is given as an infusion into a vein. A healthcare provider will give you this injection once every 3 weeks.

This medicine must be given slowly, and the infusion can take about 80 minutes to complete.

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

You may be given other medications to help prevent serious side effects or an infusion reaction.

Your doctor may have you take extra vitamin A while you are using patisiran. Take only the amount of vitamin A your doctor has prescribed. 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 vitamin A.

Patisiran doses are based on weight. Your dose needs may change if you gain or lose weight.

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 patisiran injection.

What happens if I overdose?

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

What should I avoid while using patisiran?

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

Patisiran side effects

Get emergency medical help if you have signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.

Some side effects may occur during the injection. Tell your caregiver if you feel warm, dizzy, nauseated, light-headed, or have a headache, stomach pain, chest pain, or trouble breathing.

Common side effects may include:

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 FDA at 1-800-FDA-1088.

Patisiran dosing information

Usual Adult Dose for Amyloidogenic Transthyretin Amyloidosis:

Dosing is based on actual body weight; premedication should be given at least 60 minutes prior to the start of the infusion

Weight less than 100 kg: 0.3 mg/kg via IV infusion every 3 weeks
Weight 100 kg or greater: 30 mg via IV infusion every 3 weeks

Initial infusion rate should be approximately 1 mL/min for the first 15 minutes, then increase to approximately 3 mL/min for the remainder of the infusion; in the event of an infusion related reaction, the duration of infusion may be extended

PREMEDICATION:
-Acetaminophen 500 mg orally
-IV corticosteroid (e.g., dexamethasone 10 mg or equivalent)
-IV H1 blocker (e.g. diphenhydramine 50 mg or equivalent)
-IV H2 blocker (e.g., ranitidine 50 mg or equivalent)

Comments:
-Premedications are required to reduce the risk of infusion related reactions (IRR).
-If IV premedications are not available or not tolerated, oral equivalents may be used
-If patients are tolerating the infusion, but experiencing adverse reactions to corticosteroid premedication, reduce corticosteroid in 2.5 mg increments to a minimum IV dose of dexamethasone 5 mg or equivalent
-Some patients may require additional or higher doses of one or more premedications to reduce the risk of IRRs.

Use: For the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis.

What other drugs will affect patisiran?

Other drugs may affect patisiran, 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.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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