Tryngolza▼(olezarsen) is a GalNAc3-conjugated antisense oligonucleotide inhibitor of apolipoprotein C-III (apoC-III) production1
GalNAc3-conjugate enables targeted delivery to the liver, where APOC3 mRNA is generated.1,2
ApoC-III acts on the:3
- Lipoprotein lipase (LPL)-dependent pathway by inhibiting LPL activity, the primary mechanism by which plasma triglycerides are hydrolysed3
- LPL-independent pathways by inhibiting hepatic clearance of triglyceride-rich lipoproteins, which play an important role in patients with FCS because of the substantial deficit in lipoprotein lipase (LPL) activity*3
*Hypothesised mechanism of disease.3
LPL is a key enzyme that breaks down triglycerides in the blood3,4
In genetically confirmed FCS, severely impaired LPL function forces reliance on less efficient LPL-independent pathways to break down triglycerides.3,4
FCS = familial chylomicronemia syndrome; GalNAc3 = triantennary acetylgalactosamine; mRNA = messenger ribonucleic acid; TG = triglyceride; TRL = triglyceride-rich lipoproteins; VLDL = very-low-density lipoprotein.
In genetically confirmed FCS with severely impaired LPL function, inhibition of apoC-III production frees LPL-independent pathways to break down and facilitate clearance of triglyceride-rich lipoproteins.1,3
By reducing apoC-III – a key regulator of triglyceride metabolism – Tryngolza can increase the breakdown of triglyceride-rich lipoproteins, leading to reduced triglyceride levels in the blood in people with FCS.1,3
*The efficacy and safety of olezarsen was studied in a randomised, multicentre, double-blind, placebo controlled clinical trial in adult patients with genetically confirmed FCS (N=66). Participants were randomised to receive olezarsen 80 mg (Tryngolza, n=22), olezarsen 50 mg (n=21) or placebo (n=23) once every 4 weeks for 12 months. Olezarsen 50 mg data not presented as not an approved dosing regimen for FCS.1
†Results are nominally significant (p<0.05). Given the second primary outcome of change in TG levels with olezarsen 50 mg at 6 months from baseline vs placebo did not reach significance, all secondary endpoints were considered exploratory.1
References
- Tryngolza EU Summary of Product Characteristics. December 2025.
- Stroes ESG, et al. N Engl J Med. 2024;390(19):1781–1792.
- Gaudet D, et al. N Engl J Med. 2014;371:2200–2206. doi: 10.1056/NEJMoa1400284
- Hu Y, et al. Lipids Health Dis. 2024;23(1):92. doi:10.1186/s12944-024-02086-0.
- Wang Y, et al. Expert Opin Drug Metab Toxicol. 2019;15(6):475-485.
Tryngolza▼(olezarsen) is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).
▼This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. See section 4.8 of the SmPC for how to report adverse reactions.
Healthcare professionals should report any suspected adverse reactions via their national pharmacovigilance reporting system. Suspected adverse reactions should also be reported to Sobi via email at [email protected].
This material is intended for an international audience of healthcare professionals and is based on the Tryngolza Summary of Product Characteristics (SmPC) approved for use in the European Economic Area (EEA). SmPC/Prescribing information, product availability, and pricing/reimbursement conditions may vary by country. Before prescribing, always refer to locally approved SmPC and/or prescribing information.
The Sobi Scientific Information Department is at your disposal for any additional information: [email protected].
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SOBI is a trademark of Swedish Orphan Biovitrum AB (publ).
TRYNGOLZA is a registered trademark of Arexis AB, a Sobi company, and Ionis Pharmaceuticals, Inc.
TRYNGOLZA (olezarsen) – Discovered by Ionis Pharmaceuticals, Inc.
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