Ionis Pharmaceuticals' Approved TRYNGOLZA™ for Treating Familial Chylomicronemia Syndrome
Friday, December 20, 2024
Ionis Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) approval of TRYNGOLZA™ (olezarsen) for the treatment of adults with familial chylomicronemia syndrome (FCS).
TRYNGOLZA was granted multiple designations, including Priority Review, Fast Track, Orphan Drug, and Breakthrough Therapy, reflecting its significance in addressing a critical unmet medical need. Regulatory filings for olezarsen are also ongoing for broader indications of sHTG.
This rare genetic condition, characterised by severe hypertriglyceridaemia (sHTG), can lead to life-threatening acute pancreatitis (AP).
TRYNGOLZA is now the first FDA-approved treatment specifically designed for FCS, offering significant reductions in triglyceride levels and a decreased risk of acute pancreatitis when combined with a strict low-fat diet (≤20 grams of fat per day). The treatment is self-administered via a monthly auto-injector.
The FDA’s decision follows results from the global, randomised, placebo-controlled Phase 3 Balance clinical trial, which assessed the efficacy and safety of TRYNGOLZA in adults with genetically confirmed FCS and triglyceride levels ≥880 mg/dL.
Participants receiving TRYNGOLZA demonstrated a 42.5% mean reduction in triglyceride levels compared to placebo at six months, improving to a 57% reduction at 12 months. Additionally, there was a significant decrease in acute pancreatitis episodes, with fewer patients experiencing these events compared to the placebo group.
TRYNGOLZA exhibited a favourable safety profile. Common side effects included injection site reactions, decreased platelet count, and joint pain. The findings from the Balance trial have been published in The New England Journal of Medicine (NEJM).
FCS, a rare and serious genetic condition, disrupts the body’s ability to metabolise triglycerides due to impaired lipoprotein lipase (LPL) function. This leads to triglyceride levels often exceeding 880 mg/dL, compared to normal levels below 150 mg/dL.
Patients face a high risk of acute pancreatitis, alongside symptoms such as abdominal pain, fatigue, and psychological stress. The condition affects approximately 3,000 individuals in the U.S., with many cases remaining undiagnosed.
TRYNGOLZA, an RNA-targeted treatment, works by reducing the production of apoC-III, a protein involved in triglyceride metabolism. This approval represents a milestone for both the FCS community and Ionis Pharmaceuticals, marking its transition into a commercial-stage biotechnology company.
TRYNGOLZA is expected to be available in the U.S. before the end of the year, and further regulatory reviews are underway in the European Union and other regions.
Ionis is providing support services through its Ionis Every Step™ programme, which includes insurance assistance, affordability options, and educational resources for patients and healthcare providers.
Source: ionis.com
