Volanesorsen, a product of Ionis’ proprietary antisense technology, is in development for two rare metabolic disorders: familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy (FPL). Volanesorsen is designed to reduce the production of Apolipoprotein (ApoC-III), a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters. The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted orphan drug designation to volanesorsen for the treatment of patients with FCS.
Four global trials have formed the Phase 3 program for volanesorsen.
Volanesorsen Clinical Program in FCS
Two of volanesorsen’s Phase 3 studies have included patients with FCS – the APPROACH Study in patients with FCS and the COMPASS Study in patients with severe hypertriglyceridemia. Both studies met their primary endpoints and substantially reduced triglycerides in the respective patient populations, with a 77% reduction in triglycerides in the APPROACH study. In addition, in the APPROACH study, the FCS patients with a history of high frequency pancreatitis showed a significant decrease in onstudy pancreatitis attacks when treated with volanesorsen. Together, the combined study data from APPROACH and COMPASS showed patients had a signficant reduction in new events of pancreatitis when treated with volanesorsen. In FCS, an additional Phase 3 study – the APPROACH Open Label Study – is currently ongoing.
Read more about the results of the APPROACH Study.
Following completion of the APPROACH clinical trial, the pivotal phase 3 study of volanesorsen in FCS, Akcea conducted the APPROACH Open Label Study, a multi-center study to further evaluate safety and efficacy of volanesorsen measured by percent change in fasting triglyceride levels from baseline and to assess the durability of the efficacy with extended administration of volanesorsen.
Volanesorsen Clinical Program in FPL
A Phase 3 study, called the BROADEN Study, is currently ongoing in patients with FPL.
Akcea is conducting the BROADEN Study, a randomized, placebo-controlled, double-blind Phase 2/3 clinical trial for volanesorsen in the treatment of FPL, that is currently enrolling patients. The main purpose of the BROADEN Study is to assess the effects of volanesorsen on triglyceride levels and other metabolic derangements in people with FPL.
- The study is open to men or women (≥18) with a diagnosis of FPL, type 2 diabetes and hypertriglyceridemia. Certain other inclusion and exclusion criteria apply. More information is available here.
- Participants in the BROADEN Study will be administered volanesorsen via subcutaneous injection once weekly for 52 weeks. Patients will then be eligible to participate in an open-label extension study. Following the dosing period, patients will participate in a 13-week period of follow up involving blood tests and other measurements to assess the effects of treatment with volanesorsen.
Volanesorsen Early Access Program
For information on the volanesorsen early access program, please click here.