Research & Development
Akcea and Ionis report FDA committee vote in favour of Waylivra for FCS
11 May 2018 -

An advisory committee of the US Food and Drug Administration (FDA) has voted to support approval of Waylivra (volanesorsen) for the treatment of people with familial chylomicronemia syndrome (FCS), Akcea Therapeutics Inc (NASDAQ: AKCA) and Ionis Pharmaceuticals Inc (NASDAQ: IONS) revealed on Thursday.

It comes after the committee reviewed data from two Phase 3 clinical trials, APPROACH and COMPASS, as well as the ongoing APPROACH Open Label study for Waylivra. Results from the phase 3 APPROACH trial show that patients with FCS treated with Waylivra achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased risk of pancreatitis. The most common adverse events in the APPROACH study were injection site reactions and platelet declines.

The committee's non-binding recommendation will be considered by the FDA in its review of Akcea's New Drug Application for Waylivra.

Waylivra is also under regulatory review in the European Union and Canada.

No effective therapy is currently available for FCS, an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL). The disease is characterised by severe hypertriglyceridemia and a risk of pancreatitis, which can be fatal. Patients can experience multiple severe daily and chronic symptoms, including abdominal pain and fatigue.