Study Shows That Volanesorsen Reduces Disease Burden in Patients With FCS

Akcea Therapeutics has announced the publication of results from the Re-FOCUS study that sought to assess the burden of familial chylomicronemia syndrome (FCS) on patients and the impact of Akcea's proposed drug, volanesorsen, on disease burden.

FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase. Because patients with FCS cannot break down chylomicrons, they are at risk for hypertriglyceridemia and acute, potentially fatal pancreatitis. Patients with FCS also report pain, fatigue, and imparted cognition among their physical symptoms. Currently, there is no effective therapy available to treat FCS, and patients must rely on strict dietary regimens to manage their disease, which can impact quality of life. Akcea hopes that its investigational drug, volanesorsen, can modulate multiple symptoms of the syndrome.

The new study, reported in Expert Review of Cardiovascular Therapy, was a retrospective, global, web-based survey of patients with FCS (n = 22) who received volanesorsen for 3 or more months during the APPROACH OLE study of volanesorsen for the treatment of FCS. The survey asked about patients’ experiences before and after treatment, and used a 7-point scale (on which 1 registered strong disagreement and 7 registered strong agreement) to assess patients’ perceptions and experiences.

In assessing responses, the researchers found that more patients felt that their strategies for managing their disease were effective after treatment with volanesorsen than before (40% versus 19%), and more patients felt their symptoms were well controlled with adherence to diet after treatment than before (90% versus 55%).

Patients also reported that the number of symptoms they experienced decreased with treatment; the median number of symptoms experienced was 6.5 (interquartile range [IQR], 2.0-13.0) prior to therapy, and 3.5 (IQR, 1.5-6.5) after 3 months of treatment. Patients reported reductions in the incidence of pancreatic pain, steatorrhea, physical weakness, fatigue, and back pain. Among emotional symptoms, patients reported a reduction in feelings of worry, being out of control, and anger or frustration.

When asked how much FCS impacted personal, social, and professional life, 5% of patients reported no interference from the disease before treatment, but that proportion increased to 23% after therapy. The number of patients who reported a high level of interference decreased from 59% prior to treatment to 37% after treatment, and participation in household and personal activities improved with therapy.

According to Michael W. Stevenson, RPh, PhD, global vice president of medical affairs at Akcea, “This landmark study represents an important step in our efforts to identify and quantify the many ways that FCS can affect the physical, functional and emotional health of people living with FCS. The Re-FOCUS study provides helpful, new information as we work to design and validate a tool that can accurately evaluate changes in disease burden with therapeutic intervention.”

Reference

Arca M, Hsieh A, Soran H, Rosenblit P, O’Dea L, Stevenson M. The effect of volanesoren treatment on the burden associated with familial chylomicronemia Syndrome: the results of the ReFOCUS study. [Published online June 11, 2018.] Expert Rev Cardiovasc Ther. doi: 0.1080/14779072.2018.1487290.