Patisiran

What is Patisiran?

Patisiran (ONPATTRO) contains a transthyretin-directed small interfering RNA for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis.

What are the uses of this medicine?

• This medicine is used for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

How does this medicine work?

• Patisiran is a double-stranded siRNA that causes degradation of mutant and wild-type TTR mRNA through RNA interference, which results in a reduction of serum TTR protein and TTR protein deposits in tissues.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• No clinically important drug interactions have been observed with ONPATTRO .

Is this medicine FDA approved?

• It was approved for use in the United States in 2018.

How should this medicine be used?

Recommended Dosage

• For patients weighing less than 100 kg, the recommended dosage is 0.3 mg/kg every 3 weeks by intravenous infusion. For patients weighing 100 kg or more, the recommended dosage is 30 mg.

Each of the following premedications should be given on the day of ONPATTRO infusion at least 60 minutes prior to the start of infusion:

• Intravenous corticosteroid (e.g., dexamethasone 10 mg, or equivalent)
• Oral acetaminophen (500 mg)
• Intravenous H1 blocker (e.g., diphenhydramine 50 mg, or equivalent)
• Intravenous H2 blocker (e.g., ranitidine 50 mg, or equivalent)

Administration

• ONPATTRO should be administered by a healthcare professional.
• ONPATTRO is administered via intravenous (IV) infusion. Dosing is based on actual body weight.
• If a dose is missed, administer ONPATTRO as soon as possible.
• If ONPATTRO is administered within 3 days of the missed dose, continue dosing according to the patient's original schedule.
• If ONPATTRO is administered more than 3 days after the missed dose, continue dosing every 3 weeks thereafter.
• All patients should receive premedication prior to ONPATTRO administration to reduce the risk of infusion-related reactions (IRRs).
• ONPATTRO must be filtered and diluted prior to intravenous infusion. The diluted solution for infusion should be prepared by a healthcare professional using aseptic technique.
• Infuse over approximately 80 minutes.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Lipid Complex Injection: 10 mg/5 mL (2 mg/mL) in a single-dose vial.

This medicine is available in fallowing brand namesː

• ONPATTRO

What side effects can this medication cause?

The most common side effects of this medicine include:

• upper respiratory tract infections
• infusion-related reactions

What special precautions should I follow?

• Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. Monitor for signs and symptoms during infusion. Slow or interrupt the infusion if clinically indicated. Discontinue the infusion if a serious or life-threatening infusion-related reaction occurs.
• ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplement with the recommended daily allowance of vitamin A. Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur.

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can this medicine be used in pregnancy?

• There are no available data on ONPATTRO use in pregnant women to inform a drug-associated risk of adverse developmental outcomes.
• There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to ONPATTRO during pregnancy. Physicians are encouraged to enroll pregnant patients, or pregnant women may register themselves in the program by calling 1-877-256-9526.

Can this medicine be used in children?

• Safety and effectiveness in pediatric patients have not been established.

What are the active and inactive ingredients in this medicine?

Active ingredient:

• patisiran sodium

Inactive ingredients:

• MC-3
• 1,2-DIMYRISTOYL-SN-GLYCERO-3-CARBOXAMINOPROPYLPOLYETHYLENE GLYCOL 2000 METHYL ETHER
• 1,2-Distearoyl-sn-glycero-3-phosphocholine
• Cholesterol (UNII: 97C5T2UQ7J)
• Sodium phosphate, dibasic, heptahydrate
• MONOBASIC POTASSIUM PHOSPHATE
• Sodium Chloride (UNII: 451W47IQ8X)
• Water (UNII: 059QF0KO0R)

Who manufactures and distributes this medicine?

• Packager: Alnylam Pharmaceuticals, Inc.

What should I know about storage and disposal of this medication?

• Store at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard vial if it has been frozen.
• If refrigeration is not available, ONPATTRO can be stored at room temperature up to 25°C (up to 77°F) for up to 14 days.
• For storage conditions of ONPATTRO after dilution in the infusion bag.

• Dailymed label info on Patisiran
• FDA Patisiran