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Identification
- Summary
Volanesorsen is an antisense oligonucleotide indicated to treat familial chylomicronemia.
- Brand Names
- Waylivra
- Generic Name
- Volanesorsen
- DrugBank Accession Number
- DB15067
- Background
Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA to prevent its translation.2 It is indicated to treat familial chylomicronemia, a genetic condition that prevents breakdown of triglycerides and chylomicrons.2 This drug is not commonly prescribed as it is used as an adjunct to diet in patients at high risk for pancreatitis, who have had inadequate response to triglyceride lowering therapy.2
Volanesorsen was granted a conditional approval by the European Medicines Agency.2
- Type
- Biotech
- Groups
- Approved, Investigational
- Biologic Classification
- Gene Therapies
Antisense oligonucleotides - Synonyms
- Volanesorsen
- External IDs
- ISIS 304801
Pharmacology
- Indication
Volanesorsen is conditionally approved by the EMA as an adjunct to diet in adults with familial chylomicronemia, at high risk for pancreatitis, and who have had an inadequate response to diet and triglyceride lowering therapy.2
Reduce drug development failure ratesBuild, train, & validate machine-learning modelswith evidence-based and structured datasets.Build, train, & validate predictive machine-learning models with structured datasets.- Associated Conditions
Indication Type Indication Combined Product Details Approval Level Age Group Patient Characteristics Dose Form Adjunct therapy in treatment of Familial chylomicronemia •••••••••••• ••••• •••••••••• •••••••• •• •••••••••••• •••••••• •••• •••• ••••••••••••• •••••••••• •••••••• •• •••• ••••••••• - Contraindications & Blackbox Warnings
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Volanesorsen is an antisense oligonucleotide that prevents the translation of apoC-III.1,2 It has a long duration of action as it has a half life of >2 weeks.2 Patients should be counselled regarding the risk of thrombocytopenia, raised LDL-C levels, renal toxicity, hepatotoxicity, and immunogenicity.2
- Mechanism of action
Volanesorsen is an antisense oligonucleotide that binds to apoC-III mRNA, leading to its degradation, and preventing translation of apoC-III protein.2 Normally, apoC-III would inhibit triglyceride metabolism and hepatic clearance of chylomicrons.2 Preventing translation of apoC-III allows for metabolism and breakdown of these triglycerides and chylomicrons.2
Target Actions Organism AApolipoprotein C-III inhibitorantisense oligonucleotideHumans - Absorption
Volanesorsen is approximately 80% bioavailable.2 A 285mg dose given weekly reaches a Cmax of 8.92 µg/mL, with an AUC of 136 µg*h/mL.2
- Volume of distribution
The volume of distribution at steady state is 330 L in patients with familial chylomicronemia.2
- Protein binding
Volanesorsen is >98% bound to proteins in plasma.1,2 Though the exact proteins have not been identified.1
- Metabolism
Volanesorsen is metabolized by endonucleases and exonucleases in a nonspecific manner.2 The unchanged parent drug is 26% recovered in the urine and pentamer to heptamer sized metabolites account for 55% of urinary recovery.[L16621
- Route of elimination
Volanesorsen is predominantly eliminated in the urine, though <3% of a dose was recovered within 24 hours.2
- Half-life
Volanesorsen has a half life of >2 weeks.2
- Clearance
The exact rate of clearance of volanesorsen is unknown, however it is rapidly distributed to tissues and very slowly eliminated.2
- Adverse Effects
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Data regarding overdose are not readily available.2 However, symptoms are likely to involve injection site reactions and constitutional symptomns.2 Treat patients with symptomatic and supportive care.2
- Pathways
- Not Available
- Pharmacogenomic Effects/ADRs
- Not Available
Interactions
- Drug Interactions
- This information should not be interpreted without the help of a healthcare provider. If you believe you are experiencing an interaction, contact a healthcare provider immediately. The absence of an interaction does not necessarily mean no interactions exist.Not Available
- Food Interactions
- No interactions found.
Products
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Ingredient UNII CAS InChI Key Volanesorsen sodium 6JON30SLDT 1573402-50-2 Not applicable - International/Other Brands
- Waylivra
- Brand Name Prescription Products
Name Dosage Strength Route Labeller Marketing Start Marketing End Region Image Waylivra Injection, solution 285 mg Subcutaneous Akcea Therapeutics Ireland Ltd 2020-12-16 Not applicable EU 
Waylivra Injection, solution 285 mg Subcutaneous Akcea Therapeutics Ireland Ltd 2020-12-16 Not applicable EU
Categories
- ATC Codes
- C10AX18 — Volanesorsen
- Drug Categories
- Classification
- Not classified
- Affected organisms
- Not Available
Chemical Identifiers
- UNII
- 2O4BE0K238
- CAS number
- 915430-78-3
References
- General References
- Post N, Yu R, Greenlee S, Gaus H, Hurh E, Matson J, Wang Y: Metabolism and Disposition of Volanesorsen, a 2'-O-(2 methoxyethyl) Antisense Oligonucleotide, Across Species. Drug Metab Dispos. 2019 Oct;47(10):1164-1173. doi: 10.1124/dmd.119.087395. Epub 2019 Jul 26. [Article]
- EMA Summary of Product Characteristics: Waylivra Volanesorsen Subcutaneous Injection [Link]
- External Links
- KEGG Drug
- D11648
- 2637345
- Wikipedia
- Volanesorsen
Clinical Trials
- Clinical Trials
Clinical Trial & Rare Diseases Add-on Data Package
Explore 4,000+ rare diseases, orphan drugs & condition pairs, clinical trial why stopped data, & more. Preview package Phase Status Purpose Conditions Count Start Date Why Stopped 100+ additional columns Unlock 175K+ rows when you subscribe.View sample dataNot Available No Longer Available Not Available Familial Chylomicronemia Syndrome (FCS) 1 somestatus stop reason just information to hide 3 Completed Treatment Familial Chylomicronemia Syndrome (FCS) 1 somestatus stop reason just information to hide 3 Completed Treatment Familial Chylomicronemia Syndrome (FCS) / Familial Hyperlipoproteinemia Type 1 / Lipoprotein Lipase Deficiency 1 somestatus stop reason just information to hide 3 Completed Treatment Hypertriglyceridemias 1 somestatus stop reason just information to hide 2 Completed Treatment Lipodystrophies 1 somestatus stop reason just information to hide
Pharmacoeconomics
- Manufacturers
- Not Available
- Packagers
- Not Available
- Dosage Forms
Form Route Strength Powder 1 kg/1kg Injection, solution Parenteral; Subcutaneous 285 MG Injection, solution Subcutaneous 285 mg - Prices
- Not Available
- Patents
- Not Available
Properties
- State
- Liquid
- Experimental Properties
- Not Available
Targets
- Kind
- Protein
- Organism
- Humans
- Pharmacological action
- Yes
- Actions
- InhibitorAntisense oligonucleotide
- General Function
- Component of triglyceride-rich very low density lipoproteins (VLDL) and high density lipoproteins (HDL) in plasma (PubMed:18201179, PubMed:22510806). Plays a multifaceted role in triglyceride homeostasis (PubMed:18201179, PubMed:22510806). Intracellularly, promotes hepatic very low density lipoprotein 1 (VLDL1) assembly and secretion; extracellularly, attenuates hydrolysis and clearance of triglyceride-rich lipoproteins (TRLs) (PubMed:18201179, PubMed:22510806). Impairs the lipolysis of TRLs by inhibiting lipoprotein lipase and the hepatic uptake of TRLs by remnant receptors (PubMed:18201179, PubMed:22510806). Formed of several curved helices connected via semiflexible hinges, so that it can wrap tightly around the curved micelle surface and easily adapt to the different diameters of its natural binding partners (PubMed:18408013)
- Specific Function
- cholesterol binding
- Gene Name
- APOC3
- Uniprot ID
- P02656
- Uniprot Name
- Apolipoprotein C-III
- Molecular Weight
- 10852.19 Da
References
- Post N, Yu R, Greenlee S, Gaus H, Hurh E, Matson J, Wang Y: Metabolism and Disposition of Volanesorsen, a 2'-O-(2 methoxyethyl) Antisense Oligonucleotide, Across Species. Drug Metab Dispos. 2019 Oct;47(10):1164-1173. doi: 10.1124/dmd.119.087395. Epub 2019 Jul 26. [Article]
- EMA Summary of Product Characteristics: Waylivra Volanesorsen Subcutaneous Injection [Link]
Drug created at May 20, 2019 14:46 / Updated at February 21, 2021 18:55