BOSTON, Mass. and CARLSBAD, Calif.,
The positive opinion will now be referred to the
“This positive CHMP opinion is an important step forward as we work to bring WAYLIVRA to people living with FCS who currently have no treatment options. Once approved, WAYLIVRA will be the first and only therapy for people living with the devastating challenges of FCS. We are now anticipating approval in Europe in the coming months. We will build on the strong infrastructure we have in place for TEGSEDI® (inotersen) in Europe as we prepare for the launch of WAYLIVRA,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “This is a testament to the European Regulatory Authorities’ commitment to facilitating access to innovative medicines for patients in need.”
WAYLIVRA’s Marketing Authorization Application (MAA) is based on results from the Phase 3 APPROACH study and the ongoing APPROACH Open Label Extension study and supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with WAYLIVRA reduced triglycerides by 77 percent after 3 months of treatment. The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels. In addition to the OLE study, there are also ongoing global Early Access Programs for WAYLIVRA.
“The FCS community is encouraged by the positive CHMP opinion and we remain very hopeful that people living with FCS will soon have an approved treatment available in the EU,” said Jill Prawer, founder and chair,
“WAYLIVRA will be the third medicine for which we will have received approval in the last two years. Our antisense technology platform continues to deliver many important scientific and medical advances that should support continuing growth,” said Brett P. Monia, Ph.D., chief operating officer of
ABOUT WAYLIVRA AND FCS
WAYLIVRA, a product of Ionis’ proprietary antisense technology, is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and may also affect other metabolic parameters.
WAYLIVRA is under regulatory review in the E.U. for the treatment of people with familial chylomicronemia syndrome (FCS). Akcea is working to confirm a path forward for WAYLIVRA in the US and in Canada.
FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterized by severe hypertriglyceridemia (>880mg/dL) 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% 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. There is no effective therapy for FCS currently available. Additional information on FCS is available at www.fcsfocus.com, through the LPLD Alliance at www.lpldalliance.org and through the FCS Foundation at http://www.livingwithfcs.org. For a full list of organizations supporting the FCS community worldwide, please click here.
WAYLIVRA is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea anticipates reporting top-line data from this study in mid-2019.
ABOUT AKCEA THERAPEUTICS
ABOUT IONIS PHARMACEUTICALS, INC.
As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug discovery platform has served as a springboard for actionable promise and realized hope for patients with unmet needs. We created the first and only approved treatment for children and adults with spinal muscular atrophy as well as the world's first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 novel medicines designed to treat a broad range of diseases including cardiovascular diseases, neurological diseases, infectious diseases, pulmonary diseases and cancer.
To learn more about Ionis follow us on twitter @ionispharma or visit http://ir.ionispharma.com/.
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Source: Akcea Therapeutics, Inc.
Source: Ionis Pharmaceuticals, Inc.