Press Releases

Akcea Announces FDA Advisory Committee Meeting to Review Volanesorsen as a Treatment for Familial Chylomicronemia Syndrome

February 15, 2018
Advisory Committee Meeting tentatively scheduled for May 10, 2018

CAMBRIDGE, Mass., Feb. 15, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced that the Division of Metabolism and Endocrinology Products of the U.S. Food and Drug Administration (FDA) will hold an advisory committee meeting to review data supporting the new drug application (NDA) for volanesorsen. The meeting is tentatively scheduled for May 10, 2018. Volanesorsen is an investigational therapy for the treatment of a rare lipid disorder called familial chylomicronemia syndrome (FCS), which is characterized by the buildup of fats in the body due to a deficiency in the enzyme lipoprotein lipase.

“Today, there is no effective therapy approved to treat people with FCS. Standard triglyceride-lowering agents are generally ineffective in lowering the extremely high triglyceride levels that cause the severe acute and chronic symptoms in people with FCS,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “If approved, volanesorsen would be the first therapy indicated for these patients. We look forward to reviewing the supporting data with members of the advisory committee.”

The FDA set a PDUFA (Prescription Drug User Fee Act) goal date of August 30, 2018 for the completion of its review.

ABOUT VOLANESORSEN AND FCS
Volanesorsen, 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. Volanesorsen is under regulatory review in the U.S., EU and Canada as a treatment for familial chylomicronemia syndrome (FCS). The U.S. and EU regulatory agencies have granted Orphan Drug Designation to volanesorsen for the treatment of people with FCS.

FCS is a severe, rare disorder characterized by extremely high levels of triglycerides, daily symptoms such as abdominal pain, and the risk of recurrent, potentially fatal, acute pancreatitis. People with FCS are unable to effectively metabolize large, triglyceride-rich lipid particles called chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to break down triglycerides. There is no effective therapy available. Additional information on FCS is available at www.fcsfocus.com. For a full list of organizations supporting the FCS community worldwide, please click here.

Volanesorsen 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 2019.

ABOUT AKCEA THERAPEUTICS
Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a biopharmaceutical company focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders. Akcea is advancing a mature pipeline of four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx, AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, all with the potential to treat multiple diseases. All four drugs were discovered by and are being co-developed with Ionis, a leader in antisense therapeutics, and are based on Ionis’ proprietary antisense technology. The most advanced drug in its pipeline, volanesorsen, is under regulatory review in the U.S., EU and Canada for the treatment of familial chylomicronemia syndrome, or FCS, and is currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy, or FPL. Akcea is building the infrastructure to commercialize its drugs globally with a focus on lipid specialists as the primary call point. Akcea is located in Cambridge, Massachusetts. Additional information about Akcea is available at www.akceatx.com.

FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of volanesorsen or other products in development. Any statement describing Akcea’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. Akcea’s forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea’s programs are described in additional detail in its final prospectus for its initial public offering and its most recent quarterly report on Form 10-Q, which is on file with the SEC.

In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.

Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals, Inc.

Media and Investor Contact:
Kathleen Gallagher                                                                         
Head of Communications and Investor Relations, Akcea Therapeutics
(617)-207-8509
kgallagher@akceatx.com

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Source: Akcea Therapeutics, Inc.