Ionis’ FCS drug approved in EU, launch due in Germany

Ionis Pharmaceuticals’ RNA-targeted drug Waylivra has been conditionally approved in Europe for the devastating ultra-rare disease Familial Chylomicronemia Syndrome (FCS). The European Commission’s conditional approval means Ionis and its affiliate Akcea will have to re-apply for a marketing authorisation each year for Waylivra (volanesorsen), until they can present confirmatory data to the regulator. As part of the conditional marketing authorisation, Akcea and Ionis will conduct a non-interventional post-authorisation safety study (PASS) based on data from a registry. Waylivra’s approval is based on a positive opinion from the European Medicines agency’s Committee for Medicinal Products Human Use (CHMP) earlier this year. This ran contrary to a decision from the FDA, which rejected it earlier this year, setting up a situation where Waylivra will be available in Europe but not the US. There was no immediate word from Ionis about the price of the drug, and this is likely to vary depending from one member state to the next.