Drug Profile

Obudanersen - Ionis Pharmaceuticals

Alternative Names: BIIB-121; ION-582

Latest Information Update: 27 Aug 2026

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At a glance

Originator Ionis Pharmaceuticals
Class Antisense oligonucleotides
Mechanism of Action Gene silencing; UBE3A protein expression stimulants

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

Yes - Angelman syndrome
New Molecular Entity Yes

Highest Development Phases

Phase III Angelman syndrome

Most Recent Events

24 Aug 2026 Ionis Pharmaceuticals plans a phase III trial for Angelman syndrome (In children, In adolescents, In adults) (Intrathecal) in September 2026 (NCT07782827)
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

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