Cardiology > > Dyslipidemia– Antisense oligonucleotide olezarsen slashes high triglycerides
by Kristen Monaco, Senior Staff Writer, MedPage Today December 20, 2024
The FDA authorized olezarsen (Tryngolza) as the very first drug for grownups with familial chylomicronemia syndrome, an unusual congenital disease that can trigger triglyceride levels to reach the thousands.
A first-in-class antisense oligonucleotide, olezarsen is shown in combination with a low-fat diet plan.
Approval was supported by the stage III Balance trial, which included 66 clients with serious hypertriglyceridemia as an outcome of their condition. Administered subcutaneously when a month, olezarsen at an 80-mg dosage decreased plasma triglyceride levels by 42.5 portion points compared to placebo at 6 months (95% CI -74.1 to -10.9, P=0.0084). Standard triglycerides balanced about 2,600 mg/dL (typical varieties are listed below 150 mg/dL). Olezarsen likewise resulted in a medically significant decrease in intense pancreatitis occasions.
“With no treatment choices formerly offered, we were restricted to relying just on very rigorous diet plan and way of life modifications as the sole preventative treatment choice,” stated detective Alan Brown, MD, of the Rosalind Franklin University of Medicine and Science in North Chicago, in a declaration from designer Ionis Pharmaceuticals.
“The FDA approval of Tryngolza is a crucial minute for individuals dealing with [familial chylomicronemia syndrome]their households, and doctors who now, for the very first time, have a treatment that considerably decreases triglycerides and reduces the threat of possibly dangerous intense pancreatitis occasions, as an accessory to a low-fat diet plan.”
“I am delighted to have a medication I can recommend to my clients that has actually been revealed to alter the course of their illness,” he included.
Affecting an approximated 1 to 10 per million individuals, familial chylomicronemia syndrome is an unusual type of serious hypertriglyceridemia that avoids the body from breaking down and getting rid of triglycerides from the blood stream due to an impaired function of the enzyme lipoprotein lipase. This can cause extreme stomach discomfort, xanthomas, and severe pancreatitis, which can be possibly lethal.
Olezarsen is an mRNA-targeted ligand conjugated antisense medication that works by decreasing the body’s production of apolipoprotein C-III, a crucial regulator of triglyceride metabolic process.
Typical negative occasions in the Balance trial with olezarsen consisted of injection website responses (19%), reduced platelet count (12%), and arthralgia (9%), according to the recommending info. Some cases of hypersensitivity responses, consisting of signs of bronchospasm, scattered erythema, facial swelling, urticaria, chills, and myalgias were likewise reported. Clients need to quickly look for medical attention and terminate usage if this takes place.
Olezarsen will be offered in the U.S. before completion of the year, stated Ionis.
In addition to familial chylomicronemia syndrome, the antisense oligonucleotide is likewise presently being assessed in 3 stage III trials of serious hypertriglyceridemia.
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Kristen Monaco is a senior personnel author, concentrating on endocrinology, psychiatry, and nephrology news. Based out of the New York City workplace, she’s operated at the business because 2015.
