Catalyst overview
Research summary
This catalyst tracks a PDUFA approval decision for Zilganersen (ION373) in Alexander Disease . The expected timing is based on the latest available company guidance and may change.
7/18/24- Completed enrollment in pivotal trial. 10/1/24- Granted FDA Fast Track designation. 9/22/25- Positive topline results reported. NDA submission planned in Q1 2026. 12/2/25– Received FDA Breakthrough Therapy Designation. 01/11/26:- Zilganersen EAP underway; NDA submission still planned for Q1 2026 with launch expected later in 2026. 03/23/26:- Priority Review accepted; PDUFA Sept 22, 2026. Additional pivotal data slated for AAN 2026.' 04/21/26:- Additional positive pivotal data presented at AAN 2026; PDUFA action date remains September 22, 2026.
Verification
Confirm timing and program details against the company announcement or filing.