Recently published data support long-term triglyceride (TG) and pancreatitis reduction with volanesorsen in patients with familial chylomicronaemia syndrome (FCS).1,2
In an open-label extension study of 62 patients who had previously received volanesorsen or placebo in the Phase 3 APPROACH or COMPASS studies, or were treatment-naïve, 46 completed 52 weeks and 21 completed 104 weeks treatment.1 Compared to study index baseline, volanesorsen-treated patients from the three populations had mean decreases in fasting plasma TG levels at 12, 26, 52 and 104 weeks of 48%, 55%, 50% and 50%, respectively (APPROACH); 65%, 43%, 42% and 66%, respectively (COMPASS); and 60%, 51%, 47% and 46%, respectively (treatment-naive). Common adverse events were injection site reactions and platelet count decreases, consistent with previous studies.
The VOL4002 study assessed the efficacy and safety of volanesorsen in 22 adults with FCS who were treated in the UK Early Access to Medicines Scheme (EAMS) for six to 51 months.2 Among treatment-naive patients (n=12), volanesorsen treatment resulted in an averaged median 52% reduction in TG levels at three months compared with baseline, and 47–55% reductions during 15 months of treatment Among those who had prior exposure to volanesorsen, there was a 51% reduction in TG levels from pre-treatment baseline, with reductions of 10–38% over 21 months of treatment. Pancreatitis event rates were reduced by 74% during volanesorsen treatment (one event/11 years) compared to the 5-year period before treatment (one event/2.8 years). Platelet declines were consistent with observations in Phase 3 clinical trials.
References
- Witztum JL, Gaudet D, Arca M et al. Volanesorsen and triglyceride levels in familial chylomicronemia syndrome: Long-term efficacy and safety data from patients in an open-label extension trial. J Clin Lipidol. 2023 May-Jun;17(3):342-355.
- Jones A, Peers K, Wierzbicki AS et al. Long-term effects of volanesorsen on triglycerides and pancreatitis in patients with familial chylomicronaemia syndrome (FCS) in the UK Early Access to Medicines Scheme (EAMS). Atherosclerosis. 2023 Jun;375:67-74