Patisiran

Patisiran is a prescription medication used for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR) in adults. 

• Patisiran is available under the following different brand names: Onpattro.

Common side effects of Patisiran include:

• Stuffy nose,
• Sneezing, and
• Sore throat

Serious side effects of Patisiran include:

• Hives,
• Difficulty breathing,
• Swelling of the face, lips, tongue, or throat,
• Severe dizziness,
• Warmth,
• Dizziness,
• Nausea,
• Lightheadedness,
• Headache,
• Fast heartbeats,
• Stomach pain, and
• Chest pain or pressure

Rare side effects of Patisiran include:

• None 

Seek medical care or call 911 at once if you have the following serious side effects:

• Severe headache, confusion, slurred speech, arm or leg weakness, trouble walking, loss of coordination, feeling unsteady, very stiff muscles, high fever, profuse sweating, or tremors;
• Serious eye symptoms such as sudden vision loss, blurred vision, tunnel vision, eye pain or swelling, or seeing halos around lights;
• Serious heart symptoms include fast, irregular, or pounding heartbeats; fluttering in the chest; shortness of breath; sudden dizziness, lightheadedness, or passing out.

This is not a complete list of side effects and other serious side effects or health problems that may occur because of the use of this drug. Call your doctor for medical advice about serious side effects or adverse reactions. You may report side effects or health problems to FDA at 1-800-FDA-1088.

Adult dosage

Lipid complex injection

• 2 mg/mL (5mL-vial)

Amyloidosis

Adult dosage

• Dose based on actual body weight.
• Below 100 kg: 0.3 mg/kg intravenous every 3 weeks
• Above 100 kg: 30 mg IV every 3 weeks

Dosage Considerations – Should be Given as Follows: 

• See “Dosages”

If your medical doctor is using this medicine to treat your pain, your doctor or pharmacist may already be aware of any possible drug interactions and may be monitoring you for them. Do not start, stop, or change the dosage of any medicine before checking with your doctor, health care provider, or pharmacist first.

• Patisiran has no noted severe interactions with any other drugs.
• Patisiran has no noted serious interactions with any other drugs.
• Patisiran has no noted moderate interactions with any other drugs.
• Patisiran has no noted minor interactions with any other drugs.

This information does not contain all possible interactions or adverse effects. Visit the RxList Drug Interaction Checker for any drug interactions. Therefore, before using this product, tell your doctor or pharmacist about all your products. Keep a list of all your medications with you and share this information with your doctor and pharmacist. Check with your healthcare professional or doctor for additional medical advice, health questions, or concerns.

Contraindications

• None

Effects of drug abuse

• None

Short-Term Effects

• See “What Are Side Effects Associated with Using Patisiran?”

Long-Term Effects

• See “What Are Side Effects Associated with Using Patisiran?”

Cautions

• Infusion-related reactions
  • Infusion-related reactions (IRRs) were observed in patients receiving therapy.
  • The most common symptoms reported include flushing, back pain, nausea, abdominal pain, dyspnea, and headache; severe hypotension and syncope were reported as symptoms of IRRs in the expanded access program and postmarketing setting.
  • Patients should receive premedications on the day of infusion, at least 60 min before infusion; monitor patients during the infusion for signs and symptoms of IRRs; if IRR occurs, consider slowing or interrupting the infusion and instituting medical management (. g, corticosteroids, or other symptomatic treatment), as clinically indicated (see Dosing)
  • If an infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved; in case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
  • Some patients may benefit from a slower infusion rate or additional doses of other premedications with subsequent infusions to reduce risk.
  • Severe hypotension and syncope were reported as symptoms of IRRs.
• Reduced serum vitamin A levels and recommended supplementation.
• May decrease serum vitamin A levels.
• Vitamin A supplementation is advised; higher doses than recommended daily allowance of vitamin A should not be given to try to achieve normal serum vitamin A levels during treatment; serum vitamin A levels do not reflect total vitamin A in the body.
• Refer to an ophthalmologist if a patient develops ocular symptoms of vitamin A deficiency (. g, night blindness)

Pregnancy and Lactation

• There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed during pregnancy; physicians are encouraged to enroll pregnant patients, or pregnant women may register themselves in the program by calling 1-877-256-9526 or by contacting [email protected]
• No data on use in pregnant women is available to inform a drug-associated risk of adverse developmental outcomes.
• Treatment leads to a decrease in serum vitamin A levels, and vitamin A supplementation is advised; vitamin A is essential for normal embryofoetal development; however, excessive levels of vitamin A are associated with adverse developmental effects; effects on the fetus of a reduction in maternal serum transthyretin caused by therapy and of vitamin A supplementation are unknown.
• Lactation
  • There is no information regarding the presence of the drug in human milk, its effects on the breastfed infant, or milk production.
  • Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for therapy and any potential adverse effects on the breastfed infant from therapy or the underlying maternal condition.
  • In lactating rats, patisiran was not detected in milk; however, lipid components (DLin-MC3-DMA and PEG2000-CDMG) were present in milk.