Plozasiran

Plozasiran
Clinical data
Trade namesRedemplo
Other namesARO-APOC3
AHFS/Drugs.comMonograph
MedlinePlusa626006
License data
Routes of
administration		Subcutaneous
Drug classLipid-lowering agent
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG

Plozasiran, sold under the brand name Redemplo, is a medication used for the treatment of familial chylomicronemia syndrome.[2] Plozasiran is an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA).[2] It is given by injection under the skin (subcutaneously).[2]

Plozasiran was approved for medical use in the United States in November 2025.[5] The US Food and Drug Administration considers it to be a first-in-class medication.[6][7]

Medical uses

Plozasiran is indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.[2]

Familial chylomicronemia syndrome is a rare genetic disorder that affects the body's ability to break down fats (triglycerides) in the bloodstream.[5] This leads to abnormally high levels of chylomicrons, which are particles that carry triglycerides.[5] Normal triglyceride levels are less than 150 mg/dL; levels above 500 mg/dL are considered severely high (severe hypertriglyceridemia).[5] People with familial chylomicronemia syndrome can have triglyceride levels in the thousands.[5] These high triglyceride levels can cause severe abdominal pain, inflammation of the pancreas (acute pancreatitis), and fatty deposits in the skin (xanthomas).[5] Some of these symptoms, specifically acute pancreatitis, can be life-threatening.[5]

Side effects

The most common side effects include hyperglycemia (high blood sugar), headache, nausea, and injection site reaction.[5]

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).[5] 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.[5] The primary endpoint was percent change in fasting triglycerides from baseline to month ten.[5] The median percent change in triglycerides from baseline to month ten in the plozasiran treatment group was -59% compared to the placebo group.[5]

The US Food and Drug Administration granted the application for plozasiran breakthrough therapy, orphan drug, and fast track designations.[5]

Society and culture

Plozasiran was approved for medical use in the United States in November 2025.[8]

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.[3] The applicant for this medicinal product is Arrowhead Pharmaceuticals Ireland Limited.[3][9] Plozasiran was designated an orphan drug.[10] Plozasiran was authorized for medical use in the European Union in June 2026.[3][4]

Names

Plozasiran is the international nonproprietary name.[11]

Plozasiran is sold under the brand name Redemplo.[5][8]

References

  1. ↑ "Summary Basis of Decision for Redemplo". Dhpp.hpfb-dgpsa.ca. Retrieved 14 July 2026.
  2. 1 2 3 4 5 "Redemplo- plozasiran injection, solution". DailyMed. 24 November 2025. Retrieved 28 December 2025.
  3. 1 2 3 4 "Redemplo EPAR". European Medicines Agency (EMA). 24 April 2026. Retrieved 28 May 2026. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. 1 2 "Redemplo PI". Union Register of medicinal products. 22 June 2026. Retrieved 12 July 2026.
  5. 1 2 3 4 5 6 7 8 9 10 11 12 13 14 "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. Public Domain This article incorporates text from this source, which is in the public domain.
  6. ↑ "Novel Drug Approvals for 2025". U.S. Food and Drug Administration (FDA). 8 July 2026. Retrieved 19 July 2026.
  7. ↑ "Advancing Health Through Innovation: New Drug Therapy Approvals 2025" (PDF). U.S. Food and Drug Administration (FDA). January 2026.
  8. 1 2 "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.
  9. ↑ "New medicine to reduce triglycerides in adults with familial chylomicronaemia syndrome". European Medicines Agency (EMA) (Press release). 24 April 2026. Retrieved 28 May 2026.
  10. ↑ "orphan designation for treatment of familial chylomicronaemia syndrome". European Medicines Agency (EMA). 12 November 2021. Retrieved 28 May 2026.
  11. ↑ 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

  • 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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