For the first time a drug to treat adult patients with familial chylomicronemia syndrome (FCS), a severe and rare condition that leads to extremely high level of blood fats called triglycerides, has been approved by the US Food and Drug Administration.
Plozasiran, now marketed under the brand name Redemplo, was approved based on the results of a Phase 3 study, which was published in the New England Journal of Medicine.
Winthrop Professor Gerald Watts, from The University of Western Australia's Medical School and lead author of the study, said the approval represented an important milestone for affected patients and the healthcare community.
"Extremely high blood levels of triglyceridesin the blood (referred to as chylomicronemia) are caused by a rare genetic defect known as FCS that impairs their clearance after a meal from the circulation," Professor Watts said.
"It can have serious consequences including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, fatty liver and cognitive issues.
"Before this drug was approved there were limited therapeutic options to adequately treat this condition."
The Phase 3 placebo-controlled study evaluated the efficacy and safety of plozasiran in adults with genetically confirmed or clinically diagnosed FCS.
The trial involved 75 individuals across 39 different sites in 18 countries who were randomised to receive 25mg plozasiran, 50mg plozasiran or a matching placebo by injection once every three months.
Participants who completed the randomised period were eligible to continue in a two-part extension period, where all participants received plozasiran.
"The drug was approved by the FDA as an adjunct to diet to reduce extremely high triglycerides for adults with FCS," Professor Watts said.
Plozasiran is a chemically modified small interfering ribonucleic acid (siRNA) that selectively inhibits the messenger-RNA and hepatic production of apoC-III, a protein that raises triglycerides by slowing both their breakdown by an enzyme called lipoprotein lipase, in the circulation and their clearance by receptors in the liver.
"People living with FCS have extremely high triglyceride levels and a substantially higher risk of acute pancreatitis and related long-term complications, often resulting in a reduced quality of life," Professor Watts said.
"We look forward to the requisite national approvals to enable Australians with FCS to access this important treatment."
