A drug used to treat patients with a severe and rare condition has been approved for use in the United States based off the results of a study authored by UWA professor of medicine.
The approval by the Food and Drug Administration (FDA) of plozasiran, marks the first time an siRNA (small interfering RNA) drug to treat adult patients with familial chylomicronemia syndrome (FCS), a condition that leads to extremely high level of blood fats, has been approved in the US.
Plozasiran, now marketed under the brand name Redemplo, was approved based on the results of a Phase 3 study, published in the New England Journal of Medicine.
The lead author was Winthrop Professor Gerald Watts from UWA’s medical school.
The approval was described as an important milestone for affected patients and the healthcare community.
“Extremely high blood levels of triglycerides in the blood, known as chylomicronemia, are caused by a rare genetic defect known as FCS that impairs their clearance from the circulation after a fat-containing meal,” Professor Watts said.
“It can have serious consequences, including acute and potentially fatal pancreatitis, chronic abdominal pain, skin eruptions called xanthomata, diabetes, fatty liver and cognitive issues.
“Before this drug was approved there were limited therapeutic options to adequately treat this condition.”
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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 under the skin 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 and exercise to reduce extremely high triglycerides for adults with FCS,” Professor Watts told Medical Forum.
Plozasiran is a chemically modified small interfering ribonucleic acid 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 and the clearance of their smaller remnant particles 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 poor quality of life,” Professor Watts said.
Professor Watts has been caring for patients with severe hypertriglyceridemia for 40 years. He said to have a drug that provides a much-improved quality of life was a great achievement for the patients who could access it.
In the earlier days of his career, while doctors could assist with dietary recommendations, managing a patient’s diabetes and weight using a spate or oral medication, treatment was usually ineffective.
“Many patients presented with recurrent pancreatitis and its severe disabling consequences, severe abdominal pain, pancreatic abnormalities, diabetes and persistent poor quality of life because of pain, depression, an inability to eat a normal diet at all.
“It was a very poor quality of life, with recurrent hospital admissions and long length of stays. For me, after 40 years of practice, to be able to show that one could prevent pancreatitis in this high-risk condition was a great achievement for improving outcomes in these hitherto unfortunate patients.”
The drug is available in Australia on a special access scheme for those with FCS-like characteristics, persistent chylomicronemia, a genetic defect and recurrent pancreatitis.
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