Key result
Volanesorsen is associated with ~41% lower triglycerides and zero recurrent pancreatitis in familial chylomicronemia syndrome.
Why the study?
Volanesorsen is a treatment option for familial chylomicronemia syndrome but is not approved or available in all countries, including Colombia.
Does volanesorsen reduce plasma triglyceride levels and prevent pancreatitis in patients with familial chylomicronemia syndrome?
Observational (n=10)
Does volanesorsen reduce plasma triglyceride levels and prevent pancreatitis in patients with familial chylomicronemia syndrome?
Real-world evidence from Colombia demonstrates that volanesorsen effectively reduces triglyceride levels and prevents recurrent pancreatitis in patients with familial chylomicronemia syndrome.
May inform volanesorsen use in FCS where approved; leaves open need for RCTs on long-term outcomes and access.
BACKGROUND: Familial chylomicronemia syndrome (FCS) is an ultra-rare disorder associated with pathogenic variants in genes implicated in chylomicron metabolism such as LPL, APOA5, APOC2, GPIHBP1, and LMF1. Patients with FCS have severe hypertriglyceridemia complicated with recurrent episodes of pancreatitis. Volanesorsen is a treatment option for such patients. However, this treatment is not approved or available in all countries. OBJECTIVE: To present the real-life evidence of clinical response to volanesorsen in patients with FCS in Colombia. METHODS: All patients treated with volanesorsen in Colombia as of June 25, 2024, were included. After informed consent, relevant clinical and laboratory data were obtained through review of clinical charts and records from the volanesorsen patient support program. RESULTS: Ten patients with FCS and treated with volanesorsen were included. Most cases were caused by variants in LPL. A total of 90% of cases had at least 1 episode of pancreatis, the mean number of pancreatitis episodes was 5. Median follow-up was 56.5 weeks (IQR 38.3-82.3). The median highest plasma triglyceride (TG) level before treatment was 3111 mg/dL (IQR 1738-3810), while the median lowest TG level after treatment was 493 mg/dL (IQR 147-812). The mean percent decreases in plasma TG at months 1, 3, 6, and 12 were 53.6%, 59.7%, 51.5%, and 40.5%, respectively. There were no new pancreatitis episodes after initiation of volanesorsen treatment. Side effects were consistent with those reported in clinical trials. CONCLUSION: Real-life data of volanesorsen treatment for FCS in Colombia demonstrate efficacy and safety similar to pivotal clinical trials.
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Román-Gonzaléz et al. (2025) conducted an observational in Familial chylomicronemia syndrome (FCS) (n=10). Volanesorsen was evaluated on Mean percent decrease in plasma triglycerides at 12 months. In 10 patients with familial chylomicronemia syndrome, volanesorsen treatment reduced plasma triglycerides by a mean of 40.5% at 12 months, with no new pancreatitis episodes during follow-up.
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