Ionis Pharmaceuticals rare disease drug wins FDA approval

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Ionis Pharmaceuticals received FDA approval for Tryngolza, a therapy for familial chylomicronemia syndrome (FCS), a rare genetic disease. The drug, olezarsen, was found to lower triglyceride levels and reduce the risk of pancreatitis. Ionis believes Tryngolza could also benefit patients with high triglycerides, with potential peak sales of $1.8 to $2 billion. This approval marks Ionis’ first solo drug launch in its history. Ongoing trials aim to expand the drug’s approval to treat more common conditions related to high triglycerides. Market analysts predict a strong demand for Tryngolza if approved for broader use.

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