Volanesorsen for treatment of patients with familial chylomicronemia syndrome.

Volanesorsen for treatment of patients with familial chylomicronemia syndrome. Drugs Today (Barc). 2018 Dec;54(12):721-735 Authors: Warden BA, Duell PB Abstract Familial chylomicronemia syndrome (FCS) is a rare autosomal recessive disorder typically caused by mutations in genes for lipoprotein lipase (LPL), apolipoprotein C-II (Apo-CII), apolipoprotein A-V (Apo-AV), lipase maturation factor 1 (LMF1) and glycosylphosphatidylinositol-anchored high-density lipoprotein-binding protein 1 (GPI-HBP1). FCS is associated with severe morbidity that includes recurrent pancreatitis and other problems. Effective treatment to reliably prevent complications has been unavailable, so there is a quest to identify novel interventions to achieve sustained triglyceride lowering and prevention of pancreatitis. Apolipoprotein C-III (Apo-CIII) interferes with triglyceride clearance by blocking LPL and alternative pathways. Volanesorsen is an experimental antisense oligonucleotide that inhibits translation of Apo-CIII mRNA, thereby substantially lowering plasma levels of Apo-CIII and triglycerides. It is being developed for treatment of patients with FCS and refractory hypertriglyceridemia. Data from a variety of clinical trials have been very encouraging, with documentation of excellent triglyceride-lowering efficacy, but there have been concerns about the risk of drug-related thrombocytopenia and bleeding that contributed to the recent decision by the Food...
Source: Drugs of Today - Category: Drugs & Pharmacology Tags: Drugs Today (Barc) Source Type: research