Ionis stock rises following FDA approval of TRYNGOLZA

Investing.com -- Ionis Pharmaceuticals (NASDAQ:IONS ) shares climbed 6% in pre-open trading Friday after announcing the U.S. Food and Drug Administration (FDA) approval of TRYNGOLZA™ (olezarsen) for the treatment of familial chylomicronemia syndrome (FCS), a rare and life-threatening disease. This marks a significant milestone for the company as TRYNGOLZA becomes the first approved treatment for FCS in the U.S., with Ionis expecting this to be the first of four product launches over the next three years.

TRYNGOLZA, which is to be used alongside a low-fat diet, has shown to significantly reduce triglycerides and substantially lower the risk of acute pancreatitis events in adults. The approval is based on data from the Phase 3 Balance clinical trial, which demonstrated a statistically significant reduction in triglyceride levels and a clinically meaningful decrease in acute pancreatitis events. With a favorable safety profile, the treatment is self-administered via an auto-injector once monthly.

Ionis CEO Brett P. Monia, Ph.D., hailed the FDA approval as a transformative moment for FCS patients and their families, emphasizing the treatment's potential to change the course of the disease. Ionis's evolution into a fully integrated commercial-stage biotechnology company is underscored by this approval, with a rich pipeline of medicines anticipated to follow.

The FDA's decision was influenced by the positive outcomes of the Phase 3 Balance trial, which were previously published in The New England Journal of Medicine. The trial's investigator, Dr. Alan Brown, expressed excitement over now having an effective prescription option for his FCS patients.

FCS is characterized by extremely high triglyceride levels and a high risk of acute pancreatitis, which can be fatal. In the U.S., it is estimated that FCS affects up to approximately 3,000 people, with most cases remaining undiagnosed. TRYNGOLZA will be available in the U.S. before the end of the year, and Ionis is committed to providing support services to help patients access the treatment.

Echoing the company's sentiments, TD Cowen analyst Yaron Werber commented on the approval's significance, "Today's FDA approval of Tryngolza (olezarsen; QM SC ApoCIII ASO) in familial chylomicronemia syndrome (FCS) marks a key step forward in Ionis's commercial evolution as the company's first approval of a wholly-owned, independent product. Tryngolza is now the first and only approved therapy for FCS with a label that we believe is near best case."

The approval also includes Priority Review and previously granted Fast Track, Orphan Drug, and Breakthrough Therapy designations. Additionally, olezarsen is undergoing review in the European Union, and regulatory filings in other countries are planned. Ionis is also evaluating olezarsen in three Phase 3 clinical trials for the treatment of severe hypertriglyceridemia (sHTG).

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Source: Investing.com