Ionis Pharmaceuticals and Sobi have announced the approval of Tryngolza (olezarsen) in the European Union (EU) for adults with familial chylomicronemia syndrome (FCS).
FCS is a rare genetic condition that leads to extremely high triglyceride levels, often exceeding 880 mg/dL, compared to normal levels below 150 mg/dL.
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Tryngolza is a ribonucleic acid (RNA)-targeted therapy designed to reduce the production of apoC-III, a protein involved in triglyceride metabolism.
It is already approved in the US and is also under evaluation for severe hypertriglyceridemia (sHTG), characterised by fasting triglyceride levels of 500mg/dL or higher.
The EU approval follows a favourable recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP).
Ionis CEO Brett Monia stated: “The EU approval of Tryngolza is a significant advance for the treatment of FCS.
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By GlobalData“Tryngolza has the potential to be a transformative treatment option for FCS patients in the EU who are at risk of debilitating and life-threatening acute pancreatitis attacks.
“We are proud to work with Sobi, a long-standing partner of Ionis and the FCS community, to make Tryngolza available to people with FCS in the EU.”
The EU approval is supported by data from the Phase III Balance trial, which evaluated the efficacy and safety of olezarsen in FCS patients.
The therapy showed significant reductions in fasting triglyceride levels at six months and maintained them over a year.
It also showed a significant decrease in acute pancreatitis incidents.
Results from the study, published in The New England Journal of Medicine, indicated a favourable tolerability and safety profile for Tryngolza.
In March 2025, Ionis and Sobi entered a licence agreement for the commercialisation of Tryngolza.
Sobi holds exclusive rights to market the drug outside the US, Canada and China.
