Akcea Therapeutics and Ionis Pharmaceuticals have secured conditional authorisation from the European Commission (EC) to market Waylivra (volanesorsen) for the treatment of familial chylomicronemia syndrome (FCS) in adults.
The drug is indicated as an adjunct to diet in genetically confirmed FCS patients who are at high risk for pancreatitis and had an inadequate response to diet and triglyceride reduction therapy.
EC’s conditional marketing authorisation requires the companies to perform a non-interventional post-authorisation safety study (PASS) based on a registry.
Designed by Ionis, Waylivra is an antisense oligonucleotide medicine designed to decrease the production of the ApoC-III protein that regulates plasma triglycerides.
Ionis and Akcea are co-developing the drug, which is said to be the only therapy available to treat FCS, an ultra-rare disease caused due to impaired lipoprotein lipase (LPL) function.
Akcea Therapeutics CEO Paula Soteropoulos said: “Volanesorsen is the only authorised treatment for people with FCS and is a major milestone for the global FCS community. This also marks Akcea’s second drug authorisation in the last year.”
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The EC decision is based on data from the Phase III APPROACH clinical trial and the ongoing APPROACH Open-Label Extension study. It is also supported by the Phase III COMPASS trial findings.
APPROACH results demonstrated a 77% reduction in triglycerides following three months of therapy with volanesorsen, compared to placebo.
The most common adverse events observed during the APPROACH trial were injection site reactions and reductions in platelet levels.
Furthermore, Waylivra is available through Early Access Programme (EAP) in Europe, the US and Canada. The programme has started recruiting patients.
The companies noted that the drug is associated with thrombocytopenia risk and monitoring must be carried out to ensure early detection and management of the condition.
This marketing authorisation comes after the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive recommendation for the drug in March.