Posted by Michael Wonder on 10 Sep 2025
Ionis receives US FDA breakthrough therapy designation for ION582 in Angelman syndrome
9 September 2025 - Pivotal Phase 3 REVEAL study enrollment on track to complete in 2026.
Ionis Pharmaceuticals today announced that the US FDA has granted breakthrough therapy designation to ION582 for the treatment of Angelman syndrome, a rare neurological disease that often presents in infancy and is characterised by profound intellectual disability, impaired communication, motor impairment and debilitating seizures.
