Volanesorsen effectively reduces triglycerides in severe hypertriglyceridemia, potentially decreasing the risk of acute pancreatitis, though its use is associated with thrombocytopenia.
Does volanesorsen reduce triglycerides in patients with severe hypertriglyceridemia and Familial Chylomicronemia Syndrome?
Volanesorsen is an effective treatment for reducing triglycerides in Familial Chylomicronemia Syndrome, though its use requires caution due to the risk of thrombocytopenia.
Introduction Severe hypertriglyceridemia (sHTG) is a complex disorder of lipid metabolism characterized by plasma levels of triglyceride (TG) greater than 885 mg/dl (>10 mmol/L). The treatment of sHTG syndromes is challenging because conventional treatments are often ineffective in reducing TG under the threshold to prevent acute pancreatitis (AP). The inhibition of APOC3, which encodes a protein involved in triglyceride (TG)-rich lipoproteins (TGRLs) removal, has been reported to be a novel target for the treatment of sHTG. Volanesorsen is a second-generation antisense oligonucleotide inhibiting apoC-III transcription/translation that has been recently approved in Europe for Familial Chylomicronemia Syndrome (FCS) treatment.Areas covered This review summarizes the evidences on the efficacy and safety of volanesorsen for the treatment of sHTG syndromes.Expert opinion Volanesorsen effectively reduces TG in sHTG through a mechanism that is mainly LPL-independent, potentially decreasing the risk of AP. Some safety concerns have been raised with the use of volanesorsen, mainly represented by the occurrence of thrombocytopenia. Due to the potential severity of side effects, some caution is needed before affirming the long-term utility of this drug. Despite this, volanesorsen currently remains the only drug that has been demonstrated effective in FCS, which otherwise remains an untreatable disease.
D’Erasmo et al. (2020) conducted a review in Severe hypertriglyceridemia (sHTG). Volanesorsen was evaluated. Volanesorsen effectively reduces triglycerides in severe hypertriglyceridemia, potentially decreasing the risk of acute pancreatitis, though its use is associated with thrombocytopenia.