Volanesorsen and triglyceride levels in familial chylomicronemia syndrome: Long-term efficacy and safety data from patients in an open-label extension trial
Key result
Extended volanesorsen sustains ~46% reduction in fasting triglycerides at one year in familial chylomicronemia syndrome.
Why the study?
Response to conventional TG-lowering therapies is suboptimal in FCS, prompting evaluation of the safety and efficacy of extended volanesorsen treatment.
Does extended treatment with volanesorsen reduce fasting triglyceride levels in patients with familial chylomicronemia syndrome?
Population
Patients with FCS, including previously treated participants from APPROACH and COMPASS and treatment-naive patients
Comparison
Extended volanesorsen treatment across three patient groups
Design
Phase 3 open-label extension study
Follow-up
52 weeks
Authors
Discussion
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Member takes
Implication
Supports long-term volanesorsen for sustained TG control in FCS; extends Phase 3 data on durability and consistent safety.
Structured PICO
Does extended treatment with volanesorsen reduce fasting triglyceride levels in patients with familial chylomicronemia syndrome?
P
Population
Patients with familial chylomicronemia syndrome, including those from previous trials and treatment-naive patients, evaluated over 52 weeks.
I
Intervention
Volanesorsen (extended open-label treatment)
O
Outcome
Change in fasting triglyceride (TG) and other lipid measurements, and safety over 52 weekssurrogate
Extended open-label treatment with volanesorsen provides sustained reductions in plasma triglyceride levels in patients with familial chylomicronemia syndrome.
Cite This Study
Witztum et al. (2023) studied Familial chylomicronemia syndrome (FCS). Volanesorsen was evaluated on change in fasting TG and other lipid measurements, and safety over 52 weeks. Extended open-label treatment with volanesorsen in patients with familial chylomicronemia syndrome resulted in sustained mean decreases in fasting plasma triglycerides of 42% to 50% at 12 months.