The European Medicines Agency (EMA) has recommended granting a conditional marketing authorisation for volanesorsen (Waylivra), the first medicine for the treatment of familial chylomicronaemia syndrome (FCS).
However, because of reports of severe platelet reduction with the drug, a number of risk minimisation measures are expected to be introduced, including strict dosing guidance based on regular platelet monitoring.
In the phase 3 APPROACH trial of volanesorsen in 66 patients with FCS, 52 weeks of weekly subcutaneous volanesorsen at a dose of 300 mg was associated with significant reductions in triglyceride levels.
By week 13, triglyceride levels had dropped by 77 per cent in participants in the volanesorsen group compared with an increase of 18 per cent in the placebo group (P<.0001 this effect was sustained over the treatment period ci to>
Volanesorsen-treated patients who reported at least two episodes of pancreatitis in the five years preceding randomisation suffered no attacks during the 52-week period (P=.02). A reduction in self-reported abdominal pain intensity was also observed with volanesorsen compared with placebo (P=.03).
The most common side effects were reduced platelet counts and injection site reactions. Declines in platelet counts led to five early terminations, two of which had platelets