The Medicines and Healthcare Products Regulatory Agency (MHRA) has granted Akcea Therapeutics United Kingdom, a subsidiary of Akcea Therapeutics Inc (NASDAQ: AKCA) and an affiliate of Ionis Pharmaceuticals Inc (NASDAQ: IONS), a Promising Innovative Medicine (PIM) designation for volanesorsen for the treatment of familial chylomicronaemia
syndrome (FCS), the company announced on Tuesday.
Lipoprotein lipase deficiency causes severe hypertriglyceridaemia due to chylomicronaemia, and leads to recurrent and potentially life-threatening pancreatitis.
We review the experience with familial chylomicronaemia in patients who attended the lipid clinics at Groote Schuur Hospital and Red Cross Children's War Memorial Hospital in Cape Town.
Lipaemic plasma should be taken seriously at all ages, and necessitates work-up at specialised clinics where the diagnosis of chylomicronaemia or type I hyperlipidaemia facilitates appropriate dietary management that can prevent pancreatitis.
Familial chylomicronaemia (Fredrickson type I hyperlipoproteinaemia) is a rare cause of severe hypertriglyceridaemia.
About 25% of patients with familial chylomicronaemia manifest complications before the age of 1 year, and the majority develop complications before the age of 10 years.
The records were reviewed of all patients with familial chylomicronaemia who attended the lipid clinics of Groote Schuur and/or Red Cross War Memorial Children's hospitals in Cape Town between 1984 and 2006.
Our study documents one of the largest series of patients with familial chylomicronaemia managed at a single centre.
The dyslipidaemia also manifests as a high-triglyceride/low-HDLC syndrome, and severe cases can present as a chylomicronaemia
syndrome, (33) and is associated with an increased cardiovascular risk, (36) mediated by mechanisms very similar to those postulated in diabesity.