- In January 2025, Arrowhead Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for its investigational treatment, plozasiran, for Familial Chylomicronemia Syndrome, a rare and severe genetic disorder. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 18, 2025, and has stated that it does not plan to convene an advisory committee meeting for the application.
- In December 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA (olezarsen) as an adjunct to diet for reducing triglyceride levels in adults with Familial Chylomicronemia Syndrome, a rare genetic condition causing severe hypertriglyceridemia that can result in potentially life-threatening acute pancreatitis.
- In February 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to its investigational medicine, olezarsen, for the treatment of Familial Chylomicronemia Syndrome (FCS), a rare genetic disorder marked by extremely high triglyceride levels and recurrent acute pancreatitis.
- In January 2022, PTC Therapeutics, Inc. announced that Waylivra (volanesorsen) had been granted Category 1 classification by the Câmara de Regulação do Mercado de Medicamentos (CMED), the Drug Market Regulation Chamber, in Brazil. Waylivra is the only approved treatment for Familial Chylomicronemia Syndrome (FCS) in the country.
- In August 2021, PTC Therapeutics, Inc. announced that the Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), had approved Waylivra (volanesorsen) as the first treatment for Familial Chylomicronemia Syndrome (FCS) in Brazil. FCS is a rare genetic disorder that imposes a significant disease burden on patients, including the risk of potentially fatal pancreatitis and long-term complications due to permanent organ damage.
