HQ Team
December 20, 2024: The US Food and Drug Administration has approved Ionis Pharmaceuticals’ drug to treat a rare and life-threatening genetic disease known as familial chylomicronemia syndrome (FCS), according to a statement.
The disease can lead to debilitating acute pancreatitis. It is characterised by high triglyceride levels caused by limited production of the enzyme lipoprotein lipase. An estimated 3,000 people have the disease in the US.
Ionis’s drug, olezarsen, branded as Tryngolza, was approved along with a low-fat diet and is the first-ever FDA-approved treatment for the genetic disease. The medicine is self-administered through an auto-injector once monthly.
Patients diagnosed with the disease cannot break down chylomicrons and lipoprotein particles that have 90% triglycerides — the most common type of fat in the body, making up about 95% of dietary fats.
Butter, oils
Triglycerides come from foods like butter and oils, as well as extra calories your body stores in fat cells. The body uses triglycerides for energy between meals and releases them when hormones signal it’s time.
High triglyceride levels, also known as hypertriglyceridemia, can increase your risk of heart disease, stroke, diabetes, and pancreatitis.
“For the first time, adults with FCS can now access a treatment that substantially reduces triglycerides and the risk of debilitating and potentially life-threatening acute pancreatitis,” said Brett P. Monia, PhD, chief executive officer of Ionis.
“The FDA approval of Tryngolza is also a pivotal moment for Ionis, representing our evolution into a fully integrated commercial-stage biotechnology company – a goal we set out to achieve five years ago.”
57% reduction at 12 months
The regulator’s approval was based on an end-stage trial in adult patients identified with the disease. The drug “demonstrated a statistically significant placebo-adjusted mean reduction in triglyceride levels of 42.5% from the baseline of six months.”
In 12 months, the triglyceride reductions improved further to 57%, according to the company statement.
“With no treatment options previously available, we were limited to relying only on extremely strict diet and lifestyle changes as the sole preventative treatment option,” said Alan Brown, MD, FNLA, FACC, FAHA, clinical professor of medicine, Rosalind Franklin University of Medicine and Science and trial investigator.
Lindsey Sutton Bryan, co-founder and co-president, of FCS Foundation echoed Brown’s views.
“As a rare and difficult-to-diagnose disease, FCS has a profound impact on the lives of patients and families. Many people living with FCS have experienced severe pain their whole lives – sometimes so intense they require lengthy hospitalization stays – and struggle through life with daily fatigue, nausea, brain fog and stomach pain,” she said.
Year-end availability
“Until now, our treatment options have been limited, relying on diet alone to try to manage triglyceride levels and keep acute pancreatitis attacks at bay. For the first time, adults with FCS have seen their hope for a treatment become a reality.”
Tryngolza will be available in the US before the year-end. It is undergoing review in the Europen Union and regulatory filings are planned in other nations.
The medicine is currently being evaluated in three clinical trials for the treatment of severe hypertriglyceridemia.
