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Familial chylomicronemia syndrome: Waylivra approved in the European Union

Waylivra ( Volanesorsen ) has received conditional marketing authorization from the European Commission ( EC ) as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome ( FCS ) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.
As part of the conditional marketing authorization, Akcea and Ionis will conduct a non-interventional post-authorization safety study ( PASS ) based on a Registry.

Familial chylomicronemia syndrome is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase ( LPL ) and characterized by severe hypertriglyceridemia ( more than 880mg/dL or 10mmol/L ) and a risk of unpredictable and potentially fatal acute pancreatitis.
In addition to pancreatitis, patients with familial chylomicronemia syndrome are at risk of chronic complications due to permanent organ damage, including chronic pancreatitis and pancreatogenic diabetes. They can experience daily symptoms including abdominal pain, generalized fatigue and impaired cognitions that affect their ability to work.
People with familial chylomicronemia syndrome also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog.

It is estimated that there are between 3,000 to 5,000 people living with familial chylomicronemia syndrome worldwide, with approximately 1,000 people living with familial chylomicronemia syndrome in Europe.

Waylivra is an antisense oligonucleotide drug designed to reduce the production of ApoC-III, a protein that regulates plasma triglycerides.
Waylivra is a self-administered, subcutaneous injection in a single-use, prefilled syringe.

The EC’s marketing authorization of Waylivra is based on results from the phase 3 APPROACH study and the ongoing APPROACH Open Label Extension study and is supported by results from the phase 3 COMPASS study.

Results from the APPROACH trial, the largest study ever conducted in patients with familial chylomicronemia syndrome, have shown that in comparison to placebo treatment with Waylivra delivered clinically and statistically meaningful reduction in triglycerides over the study period.
An analysis of patients with a history of recurrent pancreatitis events ( 2 events or more in the five years prior to study day 1 ) has shown a significant reduction in pancreatitis attacks in Waylivra-treated patients compared to placebo treated patients.
The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels.

Waylivra is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. ( Xagena )

Source: Akcea Therapeutics, 2019