Obudanersen - Ionis Pharmaceuticals
Alternative Names: BIIB-121; ION-582Latest Information Update: 18 Mar 2026
At a glance
- Originator Ionis Pharmaceuticals
- Class Antisense oligonucleotides
- Mechanism of Action Gene silencing; UBE3A protein expression stimulants
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Orphan Drug Status
Yes - Angelman syndrome
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Phase III Angelman syndrome
Most Recent Events
- 02 Mar 2026 Ionis Pharmaceuticals has patent protection obudanersen in USA and Europe
- 09 Sep 2025 Obudanersen receives Breakthrough Therapy status for Angelman syndrome in USA
- 01 May 2025 Phase-III clinical trials in Angelman syndrome (In adolescents, In children, In adults) in USA (Intrathecal) (NCT06914609)