FDA approves Ionis’ Tryngolza as first treatment for familial chylomicronemia syndrome
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The FDA approved Tryngolza for the treatment of adults with familial chylomicronemia syndrome when used in combination with an appropriate low-fat diet, according to an Ionis Pharmaceutical press release.
A rare genetic hyperlipidemia, familial chylomicronemia syndrome (FCS) is characterized by extremely elevated triglyceride levels and increased risk for potentially fatal recurrent acute pancreatitis, abdominal pain, hepatosplenomegaly, eruptive xanthomas, lipemia retinalis and type 3c diabetes.
Granted orphan drug designation earlier this year, Tryngolza (olezarsen) is now the first-ever FDA approved treatment for FCS capable of lowering triglyceride levels and reducing acute pancreatitis events when used in combination with a diet consisting of 20 grams of fat daily.
“The FDA approval of Tryngolza is an important moment for people living with FCS, their families and physicians who now, for the first time, have a treatment that significantly lowers triglycerides and decreases the risk of potentially life-threatening acute pancreatitis events, as an adjunct to a low-fat diet,” Alan Brown, MD, FNLA, FACC, FAHA, clinical professor of medicine at Rosalind Franklin University of Medicine and Science in North Chicago, Ill., said in the press release.
“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,” he added. “I am excited to have a medicine I can prescribe to my patients that has been shown to change the course of their disease.”
The agency based its decision on data from the phase 3 Balance trial, which assessed the efficacy and safety of Tryngolza in patients with FCS who had fasting triglyceride levels of 880 mg/dL or more at 6 and 12 months.
Results from the Balance study showed Tryngolza 80 mg statistically significantly decreased triglyceride levels from baseline to 6 months, with a placebo-adjusted mean decrease of 42.5%. At 12 months, triglyceride levels continued to drop, with a placebo-adjusted mean decrease of 57%. The drug also showed a “substantial, clinically meaningful reduction” in AP events, according to the release: Only one patient who received Tryngolza experienced a single AP event vs. seven patients from the placebo group who experienced 11 AP events.
The release stated the most common adverse events included injection site reactions, decreased platelet count and arthralgia.
“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,” Brett P. Monia, PhD, CEO of Ionis, said in the release. “We are proud of our long-standing partnership with the FCS community and are grateful to the patients, families and investigators who participated in our clinical studies, enabling Ionis to make this new treatment a reality.”
The company expects Tryngolza to be available in the U.S before the end of the year and is currently under review in the EU, with regulatory filings planned for other countries. Further, the drug is being investigated in three phase 3 trials for the treatment of severe hypertriglyceridemia.
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