Improving glycemic control in diabetic patients showing fasting
chylomicronemia will usually obviate the need for pharmacologic intervention.
Akcea Therapeutics (AKCA), an affiliate of Ionis Pharmaceuticals (IONS), announced that the final study results from the Phase 3 APPROACH study evaluating Waylivra in patients with familial
chylomicronemia syndrome were published in the August 8 issue of The New England Journal of Medicine, or NEJM.
- The US Food and Drug Administration has granted orphan drug designation to ARO-APOC3 for the treatment of familial
chylomicronemia syndrome, US-based Arrowhead Pharmaceuticals, Inc.
Therapeutic plasma exchange in patients with
chylomicronemia syndrome complicated by acute pancreatitis.
Familial
chylomicronemia syndrome (FCS) is a rare inherited lipid disorder that poses significant clinical and psychosocial burdens on patients.
The Metabolism and Endocrinology Products Advisory Committee, a division of The US Food and Drug Administration (FDA), has voted 12-8 to support approval of United States-based Akcea Therapeutics' Waylivratm (volanesorsen) intended for the treatment of familial
chylomicronemia syndrome (FCS), it was reported on Friday.
a Study of Volanesorsen (formerly IONIS-APOCIIIRx) in Patients With Familial
Chylomicronemia Syndrome (APPROACH) and the Cardiovascular Outcomes for People Using Anticoagulation Strategies (COMPASS) trial, [13] have shown a serum TG reduction of up to 50% with the use of volanesorsen, an apo C3 inhibitor, when used as monotherapy in the management of dyslipidaemia.
Although patients with a non-HDL cholesterol greater than 3.75 mmol/L require a fasting lipid profile including a triglyceride level, screening for non-fasting triglyceride can be informative since it may predict cardiovascular disease [26] and can detect
chylomicronemia leading to pancreatitis prevention [27].
My first independent scientific project was to understand why patients with lipoprotein lipase (LPL) deficiency, the
chylomicronemia syndrome, and severely increased plasma triglycerides did not develop cardiovascular disease.
Schwandt, "Treatment of primary
chylomicronemia due to familial hypertriglyceridemia by w-3 fatty acids," Metabolism, vol.
An absence of GPIHBP1 abolishes the entry of LPL into capillaries, causing severe
chylomicronemia and an accumulation of catalytically active LPL in the interstitial spaces (Voss et al., 2011).