NLA Scientific Sessions: Preview

James Radke

Later this week, Rare Disease Report will be attending the National Lipid Association (NLA) Scientific Sessions in Philadelphia, and hopes to provide the community with a variety of interest points from the meeting.
Among the highlights will be a summary of a special session on familial chylomicronemia syndrome (FCS) with Daniel Rader, MD, FNLA and Richard Dunbar, MD from the University of Pennsylvania and Daniel Gaudet, MD from the University of Montreal.
FCS is a rare, hereditary disorder, characterized by extremely high triglyceride levels and risk of pancreatitis. Patients with FCS have inadequate levels of lipoprotein lipase that helps breakdown chylomicrons. People with this condition have extremely high levels of triglycerides, up to 10 times the acceptable range. Currently, there are several drugs in development to treat FCS but at present, there no approved drugs for this condition. Patients must adhere to a strict low fat diet to reduce the risk of pancreatitis.
Last month, Seth Baum, MD of Preventive Cardiology provided an overview of the most advanced orphan drug in development for treating FCS – the antisense drug volanesorsen.
In the phase 3 APPROACH trial, 66 FCS patients received volanesorsen or placebo and the study found the volanesorsen-treated patients (n=33) achieved a statistically significant (P < .0001) mean reduction in triglycerides of 77% from baseline after 3 months of treatment, compared to a mean increase of 18% in placebo-treated patients (n=33).  Further, the reduction on triglycerides was sustained over the 52-week treatment period and none of the patients taking volanesorsen with a history of pancreatitis suffered an attack.
More details on this, and other drugs in development for FCS patients are expected to be presented at NLA Scientific Sessions.
In addition to FCS, there will also be a special session on rare lipid disorders, including discussion on:
Other rare lipidemias RDR plans to cover include LAL deficiency and HoFH.
Look for these articles from the NLA Sessions starting on Friday.
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