TRYNGOLZA demonstrated a well-tolerated safety profile in the Balance study
The safety of TRYNGOLZA was evaluated in 43 study participants who received at least 1 dose of treatment and in 23 participants who received placebo.1*
- Mean platelet counts remained within normal limits during the study, and no patient treated with TRYNGOLZA with FCS had a platelet count <25,000/mm3. There were no major bleeding events associated with a low platelet count1,2
- Adverse reactions led to discontinuation of treatment in 3 patients (7%) treated with TRYNGOLZA and 0% of patients treated with placebo. Two patients in the 80 mg TRYNGOLZA arm and 1 in the 50 mg TRYNGOLZA arm reported adverse events (diarrhea, vomiting, chest discomfort, chills, myalgia, trismus, and flushing) that led to treatment discontinuation1,2
| Total TRYNGOLZA (n=43) | Placebo (n=23) | |
|---|---|---|
| Injection-site reactions | 8 (19%) | 2 (9%) |
| Decreased platelet count | 5 (12%) | 1 (4%) |
| Arthralgia | 4 (9%) | 0 |
*The safety of TRYNGOLZA was evaluated in 66 patients with FCS enrolled in the Balance trial. In this trial, 43 patients received at least 1 dose of TRYNGOLZA, 50 mg (n=21) or 80 mg (n=22), and 23 patients received placebo. TRYNGOLZA 50 mg is not an approved dosing regimen for FCS.1
†Grouped terms composed of several similar items.1
FCS=familial chylomicronemia syndrome.
Laboratory tests1
Decrease in platelet count: TRYNGOLZA can cause reductions in platelet count. Across all trials (Balance, CORE, and CORE2), mean decreases from baseline through Week 53 ranging from 6% (50 mg, CORE and CORE2) to 10% (80 mg, across all trials) were observed in TRYNGOLZA-treated patients, whereas placebo-treated patients showed an increase of 22% in Balance or no change in CORE and CORE2. The proportion of patients experiencing a bleeding adverse event was similar between the TRYNGOLZA and placebo groups. There were no major bleeding events associated with low platelet counts.
Increase in glucose: Increases in average values in fasting glucose (≤17 mg/dL) and HbA1c (<0.2 percentage points) were observed over time with TRYNGOLZA treatment in the FCS population in Balance. The incidence of hyperglycemia (defined as adverse events, new antidiabetic medication, or laboratory values) was higher in patients with FCS treated with TRYNGOLZA without a medical history of diabetes at baseline (52%) compared with placebo-treated patients (35%).
In CORE and CORE2, increases in average values in fasting glucose (≤9 mg/dL) and HbA1c (<0.3 percentage points) were observed over time in the sHTG population treated with TRYNGOLZA. These increases were more pronounced in patients with a history of diabetes at baseline; however, increases in fasting glucose and HbA1c were also observed more frequently with TRYNGOLZA compared with placebo in patients without a history of diabetes at baseline.
Increase in liver enzymes: Mean liver enzyme values increased from baseline with TRYNGOLZA treatment but remained within the normal range. These increases occurred within the first 6 months of treatment and stabilized. However, when evaluating any liver enzyme increase in patients with sHTG, increases in liver enzymes greater than or equal to 3 times the upper limit of normal were reported more frequently with TRYNGOLZA 80 mg (7%) compared with TRYNGOLZA 50 mg (3%) and placebo (3%). Liver enzymes returned toward baseline with discontinuation of TRYNGOLZA.
Increase in hepatic fat fraction (HFF): HFF was assessed in patients with sHTG in a substudy within CORE and CORE2. Mean HFFs were elevated at baseline (17% TRYNGOLZA 50 mg, 14% TRYNGOLZA 80 mg, 14% placebo). Dose-dependent changes in HFF at 12 months of treatment were a mean increase of 2 percentage points in the TRYNGOLZA 50 mg group and 4 percentage points in the TRYNGOLZA 80 mg group vs 0 percentage points in the placebo group. The clinical meaningfulness of these findings remains uncertain.
Increase in low-density lipoprotein cholesterol (LDL-C): In all trials (Balance, CORE, and CORE2), increases were observed in LDL-C in TRYNGOLZA-treated patients compared with placebo. These increases were accompanied by decreases in non–high-density lipoprotein cholesterol (non–HDL-C). Among patients with FCS, total apolipoprotein B (apoB) increased, whereas among patients with sHTG, apoB decreased.
Balance, CORE, and CORE2 are referred to as Trial 1, Trial 2, and Trial 3, respectively, in the full Prescribing Information.1
HbA1c=hemoglobin A1c; sHTG=severe hypertriglyceridemia.