Plozasiran
CAS 2379776-40-4
2379776-41-5 SODIUM SALT
RNA, ([1′-de(6-amino-9H-purin-9-yl)]dA-(5′→5′)-sp-Am-Cm-Gm-Gm-Gm-Am-Cm-Am-(2′-deoxy-2′-fluoro)G-(2′-deoxy-2′-fluoro)U-(2′-deoxy-2′-fluoro)A-Um-Um-Cm-Um-Cm-Am-Gm-Um-Im-Am-(3′→3′)-sp-[1′-de(6-amino-9H-purin-9-yl)]dA), 3′-[O-[cis-4-[(3S,8S)-17-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]-3,8-bis[[[2-[2-[[2-(acetylamino)-2-deoxy-β-D-galactopyranosyl]oxy]ethoxy]ethyl]amino]carbonyl]-1,6,11-trioxo-15-oxa-2,7,12-triazaheptadec-1-yl]cyclohexyl] hydrogen phosphorothioate], complex with RNA (Um-sp-(2′-deoxy-2′-fluoro)C-sp-Am-sp-(2′-deoxy-2′-fluoro)C-Um-(2′-deoxy-2′-fluoro)G-Am-Gm-Am-Am-Um-(2′-deoxy-2′-fluoro)A-Cm-(2′-deoxy-2′-fluoro)U-Gm-(2′-deoxy-2′-fluoro)U-Cm-(2′-deoxy-2′-fluoro)C-Cm-(2′-deoxy-2′-fluoro)G-sp-Um) (1:1
FDA 2025, 11/18/2025, APPROVALS 2025, Redemplo, ARO-APOC3, VSA001, ARO-APOC3, VSA 001, ADS 005, XG9ARL6P25
To reduce triglycerides in adults with familial chylomicronemia syndrome
Plozasiran, sold under the brand name Redemplo, is a medication usd for the treatment of familial chylomicronemia syndrome.[1] Plozasiran is an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA).[1] It is given by injection under the skin (subcutaneously).[1]
Plozasiran was approved for medical use in the United States in November 2025.[2]
Plozasiran is under investigation in clinical trial NCT05089084 (Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)).
Plozasiran (ARO-APOC3) is an investigational RNAi therapeutic targeting apolipoprotein C-III (APOC3). It received an Orphan Drug designation by the FDA for the treatment of familial chylomicronemia syndrome.1
Plozasiran, a novel therapeutic agent, is a small interfering RNA (siRNA) developed by Silence Therapeutics. This innovative medication targets proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein involved in cholesterol metabolism, and is specifically indicated for the treatment of hypercholesterolemia, a condition characterized by elevated levels of low-density lipoprotein cholesterol (LDL-C) in the blood. Hypercholesterolemia is a significant risk factor for cardiovascular diseases, making effective treatments crucial for patient health.
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Medical uses
Plozasiran is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.[1]
Familial chylomicronemia syndrome is a rare genetic disorder that affects the body’s ability to break down fats (triglycerides) in the bloodstream.[2] This leads to abnormally high levels of chylomicrons, which are particles that carry triglycerides.[2] Normal triglyceride levels are less than 150 mg/dL; levels above 500 mg/dL are considered severely high (severe hypertriglyceridemia).[2] People with familial chylomicronemia syndrome can have triglyceride levels in the thousands.[2] These high triglyceride levels can cause severe abdominal pain, inflammation of the pancreas (acute pancreatitis), and fatty deposits in the skin (xanthomas).[2] Some of these symptoms, specifically acute pancreatitis, can be life-threatening.[2]
Side effects
The most common side effects include hyperglycemia (high blood sugar), headache, nausea, and injection site reaction.[2]
History
The efficacy of plozasiran was demonstrated in a randomized, placebo-controlled, double-blind trial (NCT05089084) in adults with genetically confirmed or clinically diagnosed familial chylomicronemia syndrome maintained on a low-fat diet (≤20 grams fat per day).[2] Participants were randomly assigned to receive four total doses of plozasiran 25 mg or matching placebo, injected subcutaneously (under the skin) once every three months over a twelve-month treatment period.[2] The primary endpoint was percent change in fasting triglycerides from baseline to month ten.[2] The median percent change in triglycerides from baseline to month ten in the plozasiran treatment group was -59% compared to the placebo group.[2]
The US Food and Drug Administration granted the application for plozasiran breakthrough therapy, orphan drug, and fast track designations.[2]
Society and culture
Legal status
Plozasiran was approved for medical use in the United States in November 2025.[3]
Names
Plozasiran is the international nonproprietary name.[4]
Plozasiran is sold under the brand name Redemplo.[2][3]
References
- https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/219947s000lbl.pdf
- “FDA approves drug to reduce triglycerides in adults with familial chylomicronemia syndrome”. U.S. Food and Drug Administration. 18 November 2025. Retrieved 21 November 2025.
This article incorporates text from this source, which is in the public domain. - “Arrowhead Pharmaceuticals Announces FDA Approval of Redemplo (plozasiran) to Reduce Triglycerides in Adults with Familial Chylomicronemia Syndrome (FCS)” (Press release). Arrowhead Pharmaceuticals. 18 November 2025. Retrieved 21 November 2025 – via Business Wire.
- World Health Organization (2024). “International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 92”. WHO Drug Information. 38 (3). hdl:10665/379650.
Further reading
- Alla SS, Shah DJ, Meyur S, Agarwal P, Alla D, Moraboina SL, et al. (2025). “Small Interfering RNA (siRNA) in Dyslipidemia: A Systematic Review on Safety and Efficacy of siRNA”. Journal of Experimental Pharmacology. 17: 249–267. doi:10.2147/JEP.S521579. PMC 12126973. PMID 40453040.
- Olatunji G, Ogieuhi IJ, Kokori E, Oluwatomiwa AV, Ajimotokan OI, Odukudu GO, et al. (November 2024). “Olezarsen and Plozasiran in Dyslipidemia Management: A Narrative Review of Clinical Trials”. High Blood Pressure & Cardiovascular Prevention. 31 (6): 567–576. doi:10.1007/s40292-024-00677-7. PMID 39352667.
- Pan Z, Zaman MA, Kalsoom S, Zhang Y (November 2024). “Messenger interference RNA therapies targeting apolipoprotein C-III and angiopoietin-like protein 3 for mixed hyperlipidemia: the future of plozasiran and zodasiran”. Expert Review of Clinical Pharmacology. 17 (11): 1017–1023. doi:10.1080/17512433.2024.2423724. PMID 39469883.
- Sydhom P, Al-Quraishi B, Gohar A, El-Shawaf M, Shehata N, Ataya M, et al. (November 2025). “The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis”. Cardiovascular Drugs and Therapy. doi:10.1007/s10557-025-07798-8. PMID 41251855.
External links
- Clinical trial number NCT05089084 for “Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) (PALISADE)” at ClinicalTrials.gov
| Clinical data | |
|---|---|
| Trade names | Redemplo |
| Other names | ARO-APOC3 |
| AHFS/Drugs.com | Redemplo |
| License data | US DailyMed: Plozasiran |
| Routes of administration | Subcutaneous |
| ATC code | None |
| Legal status | |
| Legal status | US: ℞-only[1] |
| Identifiers | |
| CAS Number | 2379776-40-4 |
| DrugBank | DB18997 |
| UNII | |
//////////Plozasiran, FDA 2025, APPROVALS 2025, Redemplo, ARO-APOC3, VSA001, ARO-APOC3, VSA 001, ADS 005, XG9ARL6P25