Akcea Therapeutics announced that it has expanded its global early access program to the US for its drug volanesorsen, which is in development for treatment of the rare lipid disorder FCS.
Today, Akcea Therapeutics announced that it has expanded its global early access program (EAP) to the United States for its drug volanesorsen, which is in development for the treatment of the rare lipid disorder familial chylomicronemia syndrome (FCS).
Volanesorsen is under regulatory review in the U.S., European Union (EU) and Canada for the treatment of FCS.
Early access, often referred to as “compassionate use,” is defined as the availability of an investigational medicinal product outside of a clinical trial that is intended to treat a serious or life-threatening condition. The EAP program is currently available in the U.S. and a number of countries in Europe; it continues to be initiated on a country-by-country basis.
“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, chief executive officer of Akcea Therapeutics in a press release. “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.”
Volanesorsen is designed specifically 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 could also potentially affect other metabolic parameters.
In a pair of recent Phase 3 trials — APPROACH and COMPASS — treatment with the drug exhibited the ability to reduce triglycerides by 77% and by 71%, respectively.
In the combined studies, incidence of pancreatitis was significantly reduced in patients treated with volanesorsen, and treatment with the drug had a mostly well-tolerated safety profile.
The most commonly reported adverse events (AE) were mild local injection site reactions. Platelet reductions did occur in both studies, but were manageable by a pause in dosing, dose reduction and treatment with corticosteroids and intravenous immunoglobulin (IVIG).
If approved, volanesorsen would be the first and only therapy indicated for people within this indication. At present, the only available therapeutic option for patients who are looking to manage symptoms associated with the condition is to follow an especially restrictive, very low-fat diet (≤20 g daily), limit simple carbohydrates, and abstain from alcohol. Even with strict adherence, triglycerides have been seen in studies to remain high, resulting in patients remaining at risk of acute pancreatitis.
Volanesorsen is also currently in Phase 3 clinical development for the treatment of patients with familial partial lipodystrophy (FPL). Akcea has stated its intentions to report top-line data from this study in 2019.