Akcea and Ionis have announced that Waylivra has received conditional marketing authorisation from the European Commission (EC) for the treatment of familial chylomicronemia syndrome (FCS).
The approval is as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, whose response to diet and triglyceride lowering therapy has been inadequate.
The authorisation follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP), and is based on results from the Phase III APPROACH study, the ongoing APPROACH Open Label Extension study and is supported by results from the Phase III COMPASS study, which showed that Waylivra delivered clinically and statistically meaningful reduction in triglycerides over the study period.
The drug is the “only treatment available for patients with FCS which makes this approval a landmark event for the global FCS community. Patients, their caretakers and their families have been suffering without any therapeutic option. Now patients across Europe can access a medicine that may help address their severely elevated triglycerides which can give them hope for better health.
“High triglycerides can lead to a multitude of severe and daily chronic symptoms such as abdominal pain and increased risk of pancreatitis which have a significant daily impact on people living with FCS,” said Jules Payne, chief executive at HEART UK and chair of FH Europe.
FCS is an ultra-rare debilitating disease that can be life-altering, caused by impaired function of the enzyme, lipoprotein lipase (LPL), which results in significant risk and disease burden, including unpredictable and potentially fatal acute pancreatitis as well as chronic complications due to permanent organ damage.