Rare Daily Staff

The European Commission (EC) granted conditional marketing authorization for Akcea Therapeutics and Ionis Pharmaceuticals Waylivra as a treatment for familial chylomicronemia syndrome (FCS), an ultra-rare, debilitating metabolic disease.

The EC granted approval to Waylivra as an adjunct to diet in adult patients with genetically confirmed FCS who are at high risk for pancreatitis and have an inadequate response to diet and triglyceride lowering therapy. As part of the conditional marketing authorization, Akcea and Ionis will be required to conduct a non-interventional post-authorization safety study based on a registry.

FCS is caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia and a risk of unpredictable and potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90 percent triglycerides. In addition to pancreatitis, FCS patients 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 FCS also report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog.

Waylivra is an antisense oligonucleotide drug designed by Ionis and co-developed by Akcea and Ionis 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.

Akcea plans to launch Waylivra in Germany this year followed by additional European countries in 2020.

“Waylivra 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,” said Jules Payne, chief executive at HEART UK and chair of FH Europe. “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.”

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 FCS, show that in comparison to a placebo treatment, Waylivra delivered a clinically and statistically meaningful reduction in triglycerides over the study period.

An analysis of patients with a history of recurrent pancreatitis events showed 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. In addition to the open label extension study, there are also ongoing global early access programs for Waylivra


Photo: Jules Payne, chief executive at HEART UK and chair of FH Europe

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