Ionis Pharmaceuticals Announces FDA Approval of TRYNGOLZA for Familial Chylomicronemia Syndrome

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announced that the U.S. Food and Drug Administration (FDA) approved TRYNGOLZA (olezarsen) as the first-ever treatment for adults with familial chylomicronemia syndrome (FCS), a rare and life-threatening condition. TRYNGOLZA, an RNA-targeted medicine, significantly reduces triglycerides and the risk of acute pancreatitis in adults with FCS when used alongside a low-fat diet. The approval was based on positive results from the Phase 3 Balance clinical trial, which demonstrated a substantial reduction in triglyceride levels and acute pancreatitis events. The drug is self-administered via an auto-injector once monthly and will be available in the U.S. before year-end.

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About Ionis Pharmaceuticals Inc.

Ionis Pharmaceuticals Inc. is a biotechnology company focused on discovering and developing RNA-targeted therapeutics. The primary mission of Ionis is to harness its expertise in antisense oligonucleotide technology to address complex genetic disorders. Ionis is pioneering in the field of RNA-targeted drug discovery, aiming to treat a wide range of diseases, including neurological, cardiovascular, metabolic, and cancer-related conditions. Notably, the company has a robust pipeline of drugs in various stages of development and partnerships with leading pharmaceutical companies to expand the reach and impact of its therapeutic discoveries. As an innovative entity in the biotechnology sector, Ionis Pharmaceuticals plays a significant role in advancing medical treatments that target the underlying genetic causes of diseases, thereby offering new hope and potential solutions to patients worldwide. Its advancements contribute substantially to the growth and evolution of personalized medicine, emphasizing the importance of genetic research in modern healthcare.