Olezarsen, sold under the brand name Tryngolza, is a

medication Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmaco ...

used in the treatment of familial chylomicronemia syndrome. It is given by

injection under the skin Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion. A subcutaneous injection is administered as a bolus into the subcutis, the layer of skin directly below the dermis and epidermis, colle ...

. Olezarsen is an

apolipoprotein C-III Apolipoprotein C-III also known as apo-CIII, and apolipoprotein C3, is a protein that in humans is encoded by the ''APOC3'' gene. Apo-CIII is secreted by the liver as well as the small intestine, and is found on triglyceride-rich lipoproteins suc ...

-directed

antisense oligonucleotide Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small fragments of nucleic aci ...

. The most common side effects include injection site reactions, low platelet counts, and joint pain. Olezarsen was approved for medical use in the United States in December 2024. The US

Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is respo ...

considers it to be a

first-in-class medication A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition. While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medic ...

Medical uses

Olezarsen is

indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ...

as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome.

Adverse effects

The most common side effects include injection site reactions, low platelet counts, and joint pain. There are some reports of allergic (hypersensitivity) reactions, including difficulty breathing, rash, facial swelling, hives, chills, and muscle aches.

Pharmacology

Olezarsen is an

-directed

. By binding to apolipoprotein C-III mRNA, it causes its degradation, which in turn increases clearance of plasma

triglycerides A triglyceride (from ''wikt:tri-#Prefix, tri-'' and ''glyceride''; also TG, triacylglycerol, TAG, or triacylglyceride) is an ester derived from glycerol and three fatty acids. Triglycerides are the main constituents of body fat in humans and oth ...

and

very low-density lipoprotein Very-low-density lipoprotein (VLDL), density relative to extracellular water, is a type of lipoprotein made by the liver. VLDL is one of the five major groups of lipoproteins (chylomicrons, VLDL, intermediate-density lipoprotein, LDL, low-density ...

(VLDL).

History

The US

(FDA) granted the application of olezarsen

orphan drug An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by th ...

designation in February 2024. In August 2024, the

European Medicines Agency The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of pharmaceutical products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products ...

granted olezarsen an orphan drug designation. The FDA approved olezarsen based on evidence from a clinical trial (trial 1; NCT04568434) of 66 participants with familial chylomicronemia syndrome. The trial was conducted at 29 sites in 11 countries including Canada, France, Italy, Netherlands, Norway, Portugal, Slovakia, Spain, Sweden, the United Kingdom, and the United States. Of the 66 participants, 19 participants were from trial sites in the United States. The benefits and side effects of olezarsen for participants with familial chylomicronemia syndrome were evaluated in the same single clinical trial. Additional trials in participants with hypertriglyceridemia were used to support the safety assessment. The number of participants representing efficacy findings may differ from the number of participants representing safety findings due to different pools of study participants analyzed for efficacy and safety. Enrolled participants were already using other treatments to lower triglycerides, including a low-fat diet and medications (such as fenofibrates, omega-3 fatty acids, and statins). Participants were randomly assigned to receive olezarsen or placebo every four weeks for one year. Neither the participants nor the health care providers knew which treatment was being given.

Society and culture

Legal status

Olezarsen was approved for medical use in the United States in December 2024..

Names

Olezarsen is the international nonproprietary name. Olezarsen is sold under the brand name Tryngolza.

References

External links

* * {{Authority control Antisense RNA Hypolipidemic agents Nucleic acids Orphan drugs Triglycerides