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Notable Approval: FDA Approves First Treatment Shown to Reduce the Risk of Acute Pancreatitis in Adults with Severe Hypertriglyceridemia
The U.S. Food and Drug Administration (FDA) has approved Tryngolza (olezarsen), used with diet, to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia.
Tryngolza is injected subcutaneously (under the skin) once per month.
Condition
Severe hypertriglyceridemia is a serious condition, defined by fasting triglyceride levels of at least 500 mg/dL. Guidelines recommend lowering triglycerides when levels are higher than 500 mg/dL to reduce the risk of acute pancreatitis (sudden inflammation of the pancreas that can be serious or life-threatening). For reference, normal triglyceride levels are less than 150 mg/dL. Standard treatment for severe hypertriglyceridemia includes lifestyle and diet modifications (such as treatment of obesity and diabetes mellitus, regular exercise, limited to no alcohol consumption, and a low-fat diet) and medications. Medications previously approved by the FDA to lower triglycerides in patients with severe hypertriglyceridemia have not had enough cases of acute pancreatitis in their trials to be able to show a reduced risk of acute pancreatitis.
Data Supporting Tryngolza
The efficacy and safety of Tryngolza were demonstrated in two randomized, double-blind, placebo-controlled trials (NCT05079919 and NCT05552326) in a total of 1,061 adults with severe hypertriglyceridemia. Across these trials, the average baseline triglyceride level was 1,116 mg/dL. The primary endpoint for both trials was the percent change in fasting triglyceride levels from baseline to month 6 (average of triglyceride levels at weeks 25 and 27) compared to placebo.
In the first trial, the average percent change in triglycerides from baseline to month 6 in the Tryngolza treatment group was -63% for the 50 mg dose and -72% for the 80 mg dose compared to the placebo group. In the second trial, the average percent change in triglycerides from baseline to month 6 in the Tryngolza treatment group was -49% for the 50 mg dose and -55% for the 80 mg dose compared to the placebo group. In the integrated analysis of the two trials, the rate of acute pancreatitis was reduced in the pooled TRYNGOLZA group compared with placebo.
Safety Information
The most common adverse reactions in patients treated with Tryngolza for severe hypertriglyceridemia are injection site reactions and liver enzyme increases. Health care providers should consider liver enzyme testing before Tryngolza initiation or an increase in dosage and when indicated thereafter. If elevations in liver enzymes persist, health care providers may consider dose interruption (a pause in taking the medication) and/or dose reduction.
Potential allergic reactions including redness of the skin, hives (red itchy bumps), swelling of the face, chills or trouble breathing have been reported in patients treated with Tryngolza. Health care providers should inform patients about the signs and symptoms of allergic reactions and instruct patients to promptly seek medical attention and discontinue the use of Tryngolza if these occur.
Designation
Tryngolza received Priority Review and Breakthrough Therapy designations for this indication. The approval was granted to Ionis Pharmaceuticals.
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