Akcea and Ionis Announce Approval of WAYLIVRA (volanesorsen) in the European Union

Akcea Therapeutics and Ionis Pharmaceuticals announced that WAYLIVRA has received conditional marketing authorization from the European Commission (EC) as an adjunct to diet in adult patients with genetically confirmed FCS and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

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.

“WAYLIVRA is the only approved treatment for people with FCS and is a major milestone for the global FCS community. This also marks Akcea’s second drug approval in the last year. We are very grateful to all the patients and physicians around the world who participated in our clinical trials. Their insights about the challenges of this devastating disease and their stories of how they have benefited from WAYLIVRA continue to motivate us,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “We plan to launch WAYLIVRA in Germany this year followed by additional European countries in 2020. Our team is ready to deliver this treatment option to patients with FCS.”

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.

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

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.

“The approval of WAYLIVRA is truly an important moment for people affected by FCS and for all of us in medicine who specialize in treating lipid disorders,” said Dr. Eric Bruckert, head of the endocrinology and prevention of cardiovascular disease department in Pitié-Salpêtrière Hospital in Paris, France. “Many clinicians are pleased to now have a treatment option available because of the significant burden of FCS on patients and their families.”

“WAYLIVRA is Ionis’ third medicine approved in just over two years. Our highly efficient and productive antisense technology has the potential to treat the untreatable by precisely targeting the root cause of disease. FCS is an example of a disease that our novel technology is ideally suited to address,” said Brett P. Monia, chief operating officer at Ionis. “We are confident that our affiliate, Akcea, has built an expert team that is committed and ready to deliver WAYLIVRA to patients as soon as possible.”

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 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 in the five years prior to Study Day 1) 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.