– – , /PRNewswire/ -- (Nasdaq: ) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for the treatment of adults with familial chylomicronemia syndrome (FCS).
The FDA has set an action date of and indicated they are not currently planning an advisory committee meeting for olezarsen. "FCS is a debilitating, rare, genetic disease that causes significant physical, emotional and financial burden with no approved treatment options in the U.S.
," said , Ph.D., chief executive officer of Ionis.
"The Priority Review underscores the urgent need people living with FCS have for a medicine that may help lower triglyceride levels and reduce incidence of life-threatening acute pancreatitis events. We look forward to partnering closely with the FDA during the review process as we work to bring this potentially breakthrough medicine to patients before the end of 2024. Separately, we are pleased with the progress of our now fully enrolled Phase 3 program investigating olezarsen in the much more common severe hypertriglyceridemia population.
" Priority Review is a designation for medicines that have the potential to provide significant improvements in the treatment, prevention or diagnosis of a serious disease, with the expectation of the FDA taking action within six months, compared to 10 months under standard review. The application to the FDA was based on po.