onpattro
Onpattro (Patisiran Sodium) is a small interfering rna used to treat Familial Amyloid Neuropathies.
Patisiran Sodium · by Alnylam Pharms Inc
Key facts
- Active ingredient
- Patisiran Sodium
- Drug class
- Small Interfering Rna
- Form
- Injectable, Lipid complex
- Strength
- Patisiran Sodium EQ 10MG BASE/5ML (EQ 2MG BASE/ML)
- Type
- Prescription (Rx)
- Brand or generic
- Brand-name
- May treat
- familial amyloid neuropathies
- Manufacturer
- Alnylam Pharms Inc
- FDA application
- NDA210922
What is Onpattro?
From the FDA label:ONPATTRO contains patisiran, a double-stranded small interfering ribonucleic acid (siRNA), formulated as a lipid complex for delivery to hepatocytes. Patisiran specifically binds to a genetically conserved sequence in the 3' untranslated region (3'UTR) of mutant and wild-type transthyretin (TTR) messenger RNA (mRNA). The structural formula is: A, adenosine; C, cytidine; G, guanosine; U, uridine; Cm, 2'- O- methylcytidine; Um, 2'- O- methyluridine; dT, thymidine ONPATTRO is supplied as a sterile, preservative-free, white to off-white, opalescent, homogeneous solution for intravenous infusion in a single-dose glass vial. Each 1 mL of solution contains 2 mg of patisiran (equivalent to 2.1 mg of patisiran sodium). Each 1 mL also contains 6.2 mg cholesterol USP, 13.0 mg (6Z,9Z,28Z,31Z)-heptatriaconta-6,9,28,31-tetraen-19-yl-4-(dimethylamino) butanoate (DLin-MC3-DMA), 3.3 mg 1,2-distearoyl- sn -glycero-3-phosphocholine (DSPC), 1.6 mg α-(3'-{[1,2-di(myristyloxy)propanoxy] carbonylamino}propyl)-ω-methoxy, polyoxyethylene (PEG 2000 -C-DMG), 0.2 mg potassium phosphate monobasic anhydrous NF, 8.8 mg sodium chloride USP, 2.3 mg sodium phosphate dibasic heptahydrate USP, and Water for Injection USP. The pH is ~7.0. The molecular formula of patisiran sodium is C 412 H 480 N 148 Na 40 O 290 P 40 and the molecular weight is 14304 Da. Chemical Structure
How to use
Onpattro is sold in more than one form (Injectable and Lipid complex), and each is dosed differently. The instructions below come from the FDA label for application NDA210922 — follow the label that came with the product you were actually prescribed.
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 ( 2.1 ) Premedicate with a corticosteroid, acetaminophen, and antihistamines ( 2.2 ) Filter and dilute prior to administration ( 2.3 ) Infuse over approximately 80 minutes ( 2.4 ) 2.1 Dosing Information ONPATTRO should be administered by a healthcare professional. ONPATTRO is administered via intravenous (IV) infusion. Dosing is based on actual body weight. For patients weighing less than 100 kg, the recommended dosage is 0.3 mg/kg once every 3 weeks. For patients weighing 100 kg or more, the recommended dosage is 30 mg once every 3 weeks. Missed Dose 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. 2.2 Required Premedication All patients should receive premedication prior to ONPATTRO administration to reduce the risk of infusion-related reactions (IRRs) [see Warnings and Precautions (5.1) ] . 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…
Side effects
The following clinically significant adverse reactions are described elsewhere in the labeling: Infusion-Related Reactions [see Warnings and Precautions (5.1) ] The most frequently reported adverse reactions (that occurred in at least 10% of ONPATTRO-treated patients and at least 3% more frequently than on placebo) were upper respiratory tract infections and infusion-related reactions ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Alnylam Pharmaceuticals at 1-877-256-9526 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical studies are conducted under widely varying conditions, adverse reaction rates observed in the clinical studies of ONPATTRO cannot be directly compared to rates in the clinical studies of another drug and may not reflect the rates observed in practice. A total of 224 patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) received ONPATTRO in the placebo-controlled and open-label clinical studies, including 186 patients exposed for at least 1 year, 137 patients exposed for at least 2 years, and 52 patients exposed for at least 3 years. In the placebo-controlled study, 148 patients received ONPATTRO for up to 18 months (mean exposure 17.7 months). Baseline demographic and disease characteristics were generally similar between treatment groups. The median age…
Warnings
Important safety information
Infusion-related reactions: 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 ( 5.1 ) Reduced serum vitamin A levels and recommended supplementation: Supplement with the recommended daily allowance of vitamin A. Refer to an ophthalmologist if ocular symptoms suggestive of vitamin A deficiency occur ( 5.2 ) 5.1 Infusion-Related Reactions Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In clinical studies, all patients received premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) to reduce the risk of IRRs. In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. Among ONPATTRO-treated patients who experienced an IRR, 79% experienced the first IRR within the first 2 infusions. The frequency of IRRs decreased over time. IRRs led to infusion interruption in 5% of patients. IRRs resulted in permanent discontinuation of ONPATTRO in less than 1% of patients in clinical studies. Across clinical studies, the most common symptoms (reported in greater than 2% of patients) of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache [see Adverse Reactions (6.1) ] . Severe hypotension and syncope have been reported as symptoms of IRRs in the expanded access program and postmarketing setting. Patients should receive premedications on the day of ONPATTRO infusion, at least 60 minutes prior to the start of infusion [see Dosage and Administration (2.2) ] . Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the ONPATTRO infusion and instituting medical management (e.g., corticosteroids or other symptomatic treatment), as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed. Some patients who experience IRRs may benefit from a slower infusion rate or additional or higher doses of one or more of the premedications with subsequent infusions to reduce the risk of IRRs [see Dosage and Administration (2.2) ] . 5.2 Reduced Serum Vitamin A Levels and Recommended Supplementation ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance of vitamin A is advised for patients taking ONPATTRO. Higher doses than the recommended daily allowance of vitamin A should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum vitamin A levels do not reflect the total vitamin A in the body. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).
Who should not take Onpattro
None. None
Drug class
- Small Interfering RNA
- Transthyretin-directed RNA Interaction
May treat (source: NIH RxClass)
See how Onpattro ranks — best-rated small interfering rna for:
Dosage forms
Injectable and Lipid complex
The forms currently marketed in the U.S., per the FDA National Drug Code Directory.
Ways to save
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Frequently asked questions
- What does Onpattro treat?
- Onpattro (Patisiran Sodium) may be used to treat familial amyloid neuropathies, based on NIH RxClass drug-classification data (conditions a drug may treat — not a verbatim copy of the FDA-approved label). This is general reference, not medical advice.
- Is there a coupon or discount for Onpattro?
- pharmaranks is an independent ratings site, not a pharmacy — we don't issue coupons or sell Onpattro. To pay less, ask your prescriber or pharmacist whether a generic equivalent exists, check the manufacturer's copay/savings card and patient-assistance program, and compare a pharmacy discount card against your insurance copay. Prices vary by pharmacy, insurance, and location, so always confirm at the counter. This is general reference, not medical or financial advice.
- Who makes Onpattro?
- Onpattro is marketed by Alnylam Pharms Inc. You can see Alnylam Pharms Inc's full profile, rating, and other products on pharmaranks.
- Is Onpattro a brand-name or generic drug?
- Onpattro is a brand-name product with the active ingredient Patisiran Sodium.
- Is Onpattro available over the counter?
- No. Onpattro is a prescription (Rx) medication in the US — you need a prescription from a licensed clinician (in person or via telehealth); it isn't sold over the counter. For some conditions there are OTC alternatives — ask your pharmacist.
- What forms does Onpattro come in?
- Onpattro is currently marketed as injectable and lipid complex, per the FDA's National Drug Code Directory.
- What class of drug is Onpattro?
- Onpattro is classified as small interfering rna, transthyretin-directed rna interaction, per the FDA's Established Pharmacologic Class.
- Is Onpattro FDA-registered?
- Onpattro is on record with the U.S. FDA under application number NDA210922. You can verify this on its official FDA label.
- Is Onpattro safe?
- There's no single safe-or-not verdict, and we don't yet have a composite recall-safety score for Onpattro. Review its FDA-label side effects and warnings below, and always consult a licensed professional.
- What are the side effects of Onpattro?
- Onpattro's side effects are taken directly from its FDA label. From the label: The following clinically significant adverse reactions are described elsewhere in the labeling: Infusion-Related Reactions [see Warnings and Precautions (5.1) ] The most frequently reported adverse reactions (that occurred in at least 10% of ONPATTRO-treated patients and at least 3% more frequently… Both common and serious reactions are documented in full on this page. This is general reference from the FDA, not medical advice — always consult a professional.
Clinical content sourced from the FDA label via openFDA (U.S. FDA). View the FDA label on DailyMed → Provided for general reference only — not medical advice. Always consult a licensed professional and the current prescribing information.
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