Lone Therapy for Rare Disease Familial Chylomicronemia Syndrome Receives Conditional Marketing Authorization in Europe

WAYLIVRA, the only therapy available for Familial Chylomicronemia Syndrome (FCS), has been authorized 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, according to a May 7 press release issued by Akcea Therapeutics, Inc. and Ionis Pharmaceuticals, Inc.

This authorization follows the positive opinion recommending approval provided by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). As part of the conditional marketing authorization, Akcea and Ionis will conduct a non-interventional post-authorization safety study (PASS) based on a Registry.

According to the release, treatment delivery will be launched in Germany in 2019 to be followed by additional European countries in 2020.

FCS is an ultra-rare debilitating disease that can be life-altering. It is 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. It is estimated that there are between 3,000 to 5,000 people living with FCS worldwide, with approximately 1,000 people living with FCS in Europe.

To read the full release, click here: https://ir.akceatx.com/news-releases/news-release-details/akcea-and-ionis-announce-approval-waylivrar-volanesorsen