Efficacy | TRYNGOLZA® (olezarsen) for FCS

TRYNGOLZA 80 mg demonstrated significant TG reduction and numerically lower incidence of AP vs placebo in FCS, the hardest-to-treat sHTG population^1-3

The primary endpoint of the Balance study was mean percent change in fasting triglycerides from baseline to Month 6 (average of Weeks 23, 25, and 27) compared with placebo.¹

43%

significant mean change in fasting triglycerides compared with placebo
(95% CI: -74, -11; P=0.0084)^‡§

57%

mean change in fasting triglycerides compared with placebo
(95% CI: -103, -11)^‡§

*Average of Weeks 23, 25, and 27.¹
^†Average of Weeks 51 and 53.⁵
^‡Difference from baseline mean fasting triglyceride levels of 2604 mg/dL.¹
^§Missing data were imputed using placebo washout imputation. The 95% CIs of treatment differences were calculated using a robust variance estimator.¹

AP=acute pancreatitis; FCS=familial chylomicronemia syndrome; sHTG=severe hypertriglyceridemia; TG=triglyceride.

Beyond triglyceride reduction, fewer AP events were observed with TRYNGOLZA 80 mg¹

Infographic

^||Adjudication based on Atlanta classification; if serum lipase and/or amylase activity was less than 3 times the upper limit of normal, imaging (preferably contrast-enhanced computed tomography) was considered to confirm the diagnosis of acute pancreatitis.⁵
^¶Time to first AP event was an ad hoc analysis.

Next See the FCS safety profile

Important safety information & Indications

IMPORTANT SAFETY INFORMATION & INDICATIONS

CONTRAINDICATIONS

TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills, and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.

Liver Enzyme Abnormalities 

TRYNGOLZA can cause increases in liver enzymes and hepatic fat in adults. Increases in liver enzymes were more frequently reported with the 80 mg dose as compared with the 50 mg dose. Consider liver enzyme testing before TRYNGOLZA initiation or an increase in dosage and when clinically indicated thereafter. If persistent elevations in liver enzymes occur, consider dose interruption and/or dose reduction. If serious hepatic injury with clinical symptoms and/or hyperbilirubinemia or jaundice occurs, promptly discontinue TRYNGOLZA.

ADVERSE REACTIONS

Adverse Reactions for FCS
Most common adverse reactions in patients with FCS (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count, and arthralgia.

Adverse Reactions for sHTG 
Most common adverse reactions in patients with sHTG (incidence ≥2% higher than placebo) were injection site reactions and liver enzyme increases.

INDICATIONS

Familial Chylomicronemia Syndrome (FCS) 
TRYNGOLZA (olezarsen) is indicated as an adjunct to diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS).

Severe Hypertriglyceridemia (sHTG) 
TRYNGOLZA is indicated as an adjunct to diet to reduce TG and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL).

Please see full Prescribing Information for TRYNGOLZA.

IMPORTANT SAFETY INFORMATION & INDICATIONS

CONTRAINDICATIONS

TRYNGOLZA is contraindicated in patients with a history of serious hypersensitivity to TRYNGOLZA or any of the excipients in TRYNGOLZA. Hypersensitivity reactions requiring medical treatment have occurred.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions

Hypersensitivity reactions (including symptoms of bronchospasm, diffuse erythema, facial swelling, urticaria, chills, and myalgias) have been reported in patients treated with TRYNGOLZA. Advise patients on the signs and symptoms of hypersensitivity reactions and instruct patients to promptly seek medical attention and discontinue use of TRYNGOLZA if hypersensitivity reactions occur.

Liver Enzyme Abnormalities

TRYNGOLZA can cause increases in liver enzymes and hepatic fat in adults. Increases in liver enzymes were more frequently reported with the 80 mg dose as compared with the 50 mg dose. Consider liver enzyme testing before TRYNGOLZA initiation or an increase in dosage and when clinically indicated thereafter. If persistent elevations in liver enzymes occur, consider dose interruption and/or dose reduction. If serious hepatic injury with clinical symptoms and/or hyperbilirubinemia or jaundice occurs, promptly discontinue TRYNGOLZA.

ADVERSE REACTIONS

Adverse Reactions for FCS
Most common adverse reactions in patients with FCS (incidence >5% of TRYNGOLZA-treated patients and >3% higher frequency than placebo) were injection site reactions, decreased platelet count, and arthralgia.

Adverse Reactions for sHTG
Most common adverse reactions in patients with sHTG (incidence ≥2% higher than placebo) were injection site reactions and liver enzyme increases.

INDICATIONS

Familial Chylomicronemia Syndrome (FCS)
TRYNGOLZA (olezarsen) is indicated as an adjunct to diet to reduce triglycerides (TG) in adults with familial chylomicronemia syndrome (FCS).

Severe Hypertriglyceridemia (sHTG)
TRYNGOLZA is indicated as an adjunct to diet to reduce TG and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG ≥500 mg/dL).

Please see full Prescribing Information for TRYNGOLZA.

References: 1. TRYNGOLZA. Prescribing information. Ionis Pharmaceuticals. 2. Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, et al; Balance Investigators. Olezarsen, acute pancreatitis, and familial chylomicronemia syndrome. N Engl J Med. 2024;390(19):1781-1792. 3. D'Erasmo L, Di Costanzo A, Cassandra F, et al. Spectrum of mutations and long-term clinical outcomes in genetic chylomicronemia syndromes. Arterioscler Thromb Vasc Biol. 2019;39(12):2531-2541. 4. Data on file. REF-01848. Ionis Pharmaceuticals; 2024. 5. Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, et al; Balance Investigators. Olezarsen, acute pancreatitis, and familial chylomicronemia syndrome. N Engl J Med. 2024;390(19)(supplementary appendix):1781-1792.