Patisiran
Pronunciation: pah-tis-er-ran
Generic name: patisiran
Brand name: Onpattro
Dosage form: solution for intravenous infusion
Drug class: Miscellaneous metabolic agents
What is patisiran?
Patisiran (brand name Onpattro) is an injectable treatment for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) that is administered by a healthcare provider once every 3 weeks.
- Patisiran is given via intravenous infusion over about 80 minutes.
Patisiran works by breaking down mutant and wild-type transthyretin (TTR) proteins by targeting their RNA and is called a transthyretin-directed small interfering RNA molecule. People with hATTR have a mutant gene that produces abnormal TTR and patisiran's mechanism of action means it decreases serum levels of TTR protein and protein deposits in tissues, relieving the symptoms of polyneuropathy.
Patisiran was FDA-approved on August 10, 2018, under the brand name Onpattro. There is no Onpattro generic.
Patisiran side effects
The most common side effects of patisiran include:
- upper respiratory tract infections (with symptoms such as stuffy nose, sneezing, and sore throat)
- infusion-related reactions.
Serious side effects and warnings
Patisiran can cause the following serious side effects.
Infusion-related reactions. A reaction may occur while you are being administered your infusion of patisiran, in some people this could be serious or life-threatening. This reaction is more likely to occur during your first 2 infusions of patisiran, although it can occur at any time. Your healthcare provider will give you premedications before your infusion to try to reduce your risk. 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.
Patisiran 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.
Get emergency medical help if you have signs of an allergic reaction to patisiran with symptoms such as hives; difficulty breathing; and swelling of your face, lips, tongue, or throat.
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.
Before receiving patisiran
Before receiving patisiran tell your doctor about all your medical conditions or allergies. Especially tell your doctor if you:
- have allergies to patisiran, Onpattro, or any of the inactive ingredients in the vial
- have experienced infusion-related reactions to medications in the past
- have a cold, the flu, or a respiratory tract infection
- have low vitamin A levels
- are pregnant or intending to become pregnant
- are breastfeeding or intend to breastfeed.
Pregnancy
Patisiran 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 patisiran on the baby.
If you are receiving patisiran and are told to take vitamin A and you are pregnant, take only the amount of vitamin A your doctor has prescribed. Vitamin A deficiency or excess vitamin A in expectant mothers can cause vision problems or CNS deformities and other serious side effects in the unborn baby.
Breastfeeding
It may not be safe to breastfeed while using this medicine. Ask your doctor about any risks.
How is patisiran administered?
Patisiran is given by a healthcare provider into a vein (intravenously) over about 80 minutes.
- It is usually given once every 3 weeks.
- 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 any burning, pain, or swelling around the IV needle when patisiran is injected.
Your doctor may tell you to take vitamin A while receiving patisiran.
- Take only the amount of vitamin A your doctor has prescribed, especially if you are pregnant.
- Vitamin A deficiency or excess in expectant mothers can cause vision problems or CNS deformities and other serious side effects in the unborn baby.
- Call your doctor at once if you have vision problems (especially at night) while receiving patisiran.
You may need frequent medical tests while receiving patisiran including frequent eye examinations.
Patisiran dosing information
The recommended dosage of patisiran is based on your weight.
- Weight less than 100 kg: patisiran 0.3 mg/kg every 3 weeks.
- Weight of 100 kg or more: patisiran 30 mg every 3 weeks.
- Your dosage may change if you gain or lose weight.
What happens if I miss a dose?
Call your doctor for instructions if you miss an appointment for your patisiran.
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.
Avoid taking more vitamin A with patisiran than your healthcare provider has prescribed.
What other drugs will affect patisiran?
Other drugs interact with 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.
No formal clinical drug interaction studies have been performed but patisiran is not expected to cause drug-drug interactions or to be affected by inhibitors or inducers of cytochrome P450 enzymes.
Do not take more vitamin A with patisiran than your healthcare provider has prescribed.
See the patisiran prescribing information for more information about drug interactions.
Storage
Your healthcare provider will store patisiran 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, patisiran 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 for intravenous infusion.
Who makes patisiran?
Alnylam Pharmaceuticals, Inc. manufactures patisiran under the brand name Onpattro. There is no patisiran generic.
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
More FAQ
References
More about patisiran
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- Drug class: miscellaneous metabolic agents
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