Vertex Pharmaceuticals Inc. (VRTX) recently released results from its Phase 2 study of suzetrigine, an investigational oral investigational medication for the treatment of patients with urea cycle disorders (UCDs). The open-label study evaluated the safety, efficacy, and pharmacodynamics of suzetrigine in patients with UCDs, a group of genetic disorders that affect the body’s ability to remove waste products from the blood.
The study found that suzetrigine significantly reduced blood ammonia levels, a key biomarker of UCDs, from a mean baseline of 83.6 μmol/L to 28.4 μmol/L at week 16. Additionally, 71.4% of patients achieved a 30% reduction in blood ammonia levels, and 50.9% achieved a 50% reduction. The most common adverse events (AEs) were headache, fatigue, and diarrhea. The study also found that suzetrigine was generally well-tolerated, with no serious AEs related to the investigational drug.
These results suggest that suzetrigine may offer a new treatment option for patients with UCDs, which are often characterized by high levels of blood ammonia and Associated neurological complications. The study will be presented at the American Academy of Neurology (AAN) 2023 Annual Meeting.