Ionis Pharmaceuticals

Ionis Pharmaceuticals got FDA approval for Tryngolza (olezarsen), the first treatment for familial chylomicronemia syndrome. Shares are undervalued, trading in 5-star territory.

FDA approves Tryngolza (olezarsen) to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS). Tryngolza, developed by Ionis Pharmaceuticals, is a subcutaneous RNA-targeted therapy designed to lower apoC-III production, a key regulator of triglyceride metabolism. It is the first medication approved for FCS, with a price of $595,000. The approval was based on the phase 3 Balance clinical trial, showing significant reductions in triglyceride levels and acute pancreatitis events.

FDA approves TRYNGOLZA™ (olezarsen) as the first treatment for FCS, reducing triglycerides and acute pancreatitis events in adults with FCS, a rare genetic disease. TRYNGOLZA is self-administered monthly and is the first of four planned launches by Ionis Pharmaceuticals.

Ionis Pharmaceuticals' Tryngolza is the first FDA-approved treatment for familial chylomicronemia syndrome (FCS). FDA senior advisor Robert Temple to retire. Zealand Pharma's glepaglutide for short bowel syndrome rejected, requiring an additional trial. Sangamo grants Astellas rights to use a viral capsid for gene therapy delivery. BioAge Labs partners with Novartis to discover drug targets for age-related illnesses.

FDA approves Ionis Pharmaceuticals' TRYNGOLZA (olezarsen) for reducing triglyceride levels in adults with familial chylomicronaemia syndrome (FCS), significantly lowering acute pancreatitis events. This is the first FDA-approved therapy for FCS, offering a monthly self-administered treatment.

Ionis Pharmaceuticals' shares rose 5% after FDA approved its RNA-targeted therapy, Tryngolza, for familial chylomicronemia syndrome (FCS), marking the first treatment for FCS in the U.S. The approval is supported by phase III BALANCE study results showing significant triglyceride-lowering and reduced acute pancreatitis attacks. Tryngolza is Ionis' first wholly-owned drug and independent commercial launch, expected by year-end. The company is also evaluating Tryngolza for severe hypertriglyceridemia and has collaborations with major drugmakers for various treatments.

The FDA approved Ionis Pharmaceuticals' Tryngolza, the first treatment for familial chylomicronemia syndrome, targeting apolipoprotein C-III mRNA to reduce triglyceride levels. Tryngolza, to be available by year-end, showed a 42.5% mean reduction in triglycerides and reduced acute pancreatitis events in a Phase III study. The approval marks a significant development for FCS patients, though market potential is limited due to the rare disease's prevalence.

Joseph Stringer of Needham maintains a Buy rating on Ionis Pharmaceuticals with a $60 target, citing FDA approval of TRYNGOLZA for FCS treatment and its market potential. Citi also rates it Buy, with a $67 target. Ionis focuses on antisense technology for drug development.