Akcea Therapeutics, Inc., a biopharmaceutical company,, has expanded its global early access programme (EAP) to the United States for people with familial chylomicronemia syndrome (FCS). Volanesorsen is under regulatory review in the United States (US), European Union (EU) and Canada for the treatment of FCS. Akcea, an affiliate of Ionis Pharmaceuticals, Inc., is focused on developing and commercialising drugs to treat patients with serious and rare diseases. Programme currently available in the US and select countries in Europe; initiating additional countries throughout 2018

Early access, also called “compassionate use,” is the availability of an investigational medicinal product 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 programme to people who may benefit while our regulatory applications are being reviewed,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics. “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 EAP programme is currently available in the US and certain countries in Europe. It continues to be initiated on a country-by-country basis globally.

Volanesorsen, a product of Ionis’ proprietary antisense technology, is under regulatory review in the US, EU and Canada as a treatment for familial chylomicronemia syndrome (FCS). The US and EU regulatory agencies have granted Orphan Drug Designation to volanesorsen for the treatment of FCS. If approved, volanesorsen would be the first and only therapy indicated for people with FCS.

FCS is an under-recognised disease caused by impaired function of the enzyme lipoprotein lipase (LPL) and characterised 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 and can experience daily symptoms including abdominal pain, generalised fatigue and impaired cognitions that affect their ability to work and also often report major emotional and psychosocial effects including anxiety, social withdrawal, depression and brain fog. There is no effective therapy for FCS currently available.

Volanesorsen 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 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.