The Phase 3 APPROACH study of the drug volanesorsen met its primary endpoint of reducing fat molecule (triglyceride) levels in patients with familial chylomicronemia syndrome (FCS), a condition that puts patients at risk of recurrent and potentially fatal pancreatitis.
The study found that triglyceride levels reduced on average by 77% after three months of treatment.
Patients with the highest documented frequency of pancreatitis attacks suffered no such attacks during the 52-week treatment period, Ionis revealed.
A reduction in abdominal pain was also observed by those on the volanesorsen treatment.
“We are excited about the strong profile of volanesorsen in not only robustly reducing triglycerides, but also providing additional important patient benefits. FCS is a life-threatening, rare disease with multiple severe daily and chronic manifestations. We believe the efficacy and safety data from volanesorsen studies demonstrate a favorable risk-benefit profile for patients with FCS,” said Paula Soteropoulos, president and chief executive officer, Akcea Therapeutics, a subsidiary of Ionis.
Dr Louis O’Dea, Akcea’s chief medical officer, said the success of the APPROACH study represented an important milestone towards the company’s first regulatory submissions for volanesorsen in the US, Canada and Europe in 2017.
“We seek to bring this new treatment as expeditiously as possible to FCS patients who have a high unmet need with potentially life-threatening consequences,” Dr O’Dea said.
Story by ProactiveInvestors