The U.S. Food and Drug Administration (FDA) has approved Ionis Pharmaceuticals’ drug, olezarsen (branded as Tryngolza), to treat familial chylomicronemia syndrome (FCS), a rare genetic disorder. This marks the company’s first wholly-owned drug.
FCS is caused by a deficiency in the lipoprotein lipase enzyme, which is responsible for breaking down triglycerides. This leads to extremely high triglyceride levels in the blood, causing inflammation of the pancreas. Currently, a strict low-fat diet is the first-line treatment, but olezarsen is now approved as an add-on therapy to help reduce triglyceride levels in adults with the condition.
The drug showed a 44% reduction in triglyceride levels compared to a placebo in late-stage trials, though patients are still required to follow dietary restrictions. FCS affects fewer than 5,000 people in the U.S.
Ionis’ CEO, Brett Monia, stated the company has already manufactured the drug and is prepared for distribution. He also indicated the drug would be priced as a first-time treatment for a severe, rare disease, with details to be shared during an investor call.
Although the disorder is rare and a competitor drug is expected next year, BMO Capital Markets analyst Kostas Biliouris expects peak sales of $341 million for the drug in FCS.
Ionis has partnered with other major drugmakers, such as Biogen and Novartis, to develop additional treatments.
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