Medicine:Plozasiran
Plozasiran, sold under the brand name Redemplo, is a medication used 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]
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]
In April 2026, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Redemplo, intended for the treatment of adults with familial chylomicronaemia syndrome.[4] The applicant for this medicinal product is Arrowhead Pharmaceuticals Ireland Limited.[4][5] Plozasiran was designated an orphan drug.[6]
Names
Plozasiran is the international nonproprietary name.[7]
Plozasiran is sold under the brand name Redemplo.[2][3]
References
- ↑ 1.0 1.1 1.2 1.3 Cite error: Invalid
<ref>tag; no text was provided for refs namedRedemplo FDA label - ↑ 2.00 2.01 2.02 2.03 2.04 2.05 2.06 2.07 2.08 2.09 2.10 2.11 2.12 2.13 "FDA approves drug to reduce triglycerides in adults with familial chylomicronemia syndrome". 18 November 2025. https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-drug-reduce-triglycerides-adults-familial-chylomicronemia-syndrome.
This article incorporates text from this source, which is in the public domain.
- ↑ 3.0 3.1 "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.
- ↑ 4.0 4.1 "Redemplo EPAR". 24 April 2026. https://www.ema.europa.eu/en/medicines/human/EPAR/redemplo. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome". European Medicines Agency (EMA) (Press release). 24 April 2026. Retrieved 28 May 2026.
- ↑ "orphan designation for treatment of familial chylomicronaemia syndrome". 12 November 2021. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2459.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 92". WHO Drug Information 38 (3). 2024.
Further reading
- "Small Interfering RNA (siRNA) in Dyslipidemia: A Systematic Review on Safety and Efficacy of siRNA". Journal of Experimental Pharmacology 17: 249–267. 2025. doi:10.2147/JEP.S521579. PMID 40453040.
- "Olezarsen and Plozasiran in Dyslipidemia Management: A Narrative Review of Clinical Trials". High Blood Pressure & Cardiovascular Prevention 31 (6): 567–576. November 2024. doi:10.1007/s40292-024-00677-7. PMID 39352667.
- "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. November 2024. doi:10.1080/17512433.2024.2423724. PMID 39469883.
- "The Efficacy and Safety of Plozasiran on Lipid Profile in Dyslipidemic Disorders: A Systematic Review and Meta-Analysis". Cardiovascular Drugs and Therapy. November 2025. 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
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