Rare Daily Staff
Akcea Therapeutics said it has expanded its global early access program to the United States for its drug volanesorsen, which is in development for familial chylomicronemia syndrome, a rare lipid disorder.
FCS is a severe condition where patients have extremely high levels of triglycerides in their blood, causing a range of symptoms including potentially fatal attacks of acute pancreatitis. Patients cannot effectively metabolize triglyceride-rich lipid particles called chylomicrons due to a deficiency in the enzyme lipoprotein lipase, which helps break down triglycerides.
Volanesorsen is an antisense drug developed by Ionis Pharmaceuticals. It 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. It is under regulatory review in the United States, European Union, and Canada for the treatment of FCS. If approved, volanesorsen would be the first and only therapy indicated for people with FCS.
Early access, also known as “compassionate use,” is the availability of an experimental therapy outside of a clinical trial that is intended to treat a serious or life-threatening condition.
“Our work is driven by a steadfast commitment to the FCS community, and we are pleased to make volanesorsen available through our global early access program to people who may benefit while our regulatory applications are being reviewed,” said Paula Soteropoulos, CEO of Akcea. “FCS is a serious condition with a variety of symptoms, including potentially fatal acute pancreatitis, that can severely impact quality-of-life. As there are no currently approved treatments for FCS, we are working closely with lipid specialists to responsibly enable patient access.”
The early access program is currently available in the United States and certain countries in Europe. It continues to be initiated on a country-by-country basis globally.
Physicians who are interested in learning more about the early access program for their patients should contact [email protected].
May 2, 2018
Photo: Paula Soteropoulos, CEO of Akcea
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