ION582, a new medicine for Angelman Syndrome, received FDA Breakthrough status! It aims to fix the root cause, showing early promise in improving communication, thinking, and movement for this severe neurological condition.
Ionis's olezarsen (Tryngolza) shows "unprecedented" results for severe hypertriglyceridemia, cutting triglycerides by up to 72% and acute pancreatitis events by 85% in Phase 3 trials—a first! Favorable safety. sNDA planned by year-end.
The FDA accepted non-invasive Liver Stiffness Measurement as a surrogate endpoint for MASH clinical trials, replacing biopsies. This key decision accelerates MASH drug development by enabling safer monitoring, speeding recruitment, and predicting patient outcomes.
