Drug Profile

Opemalirsen - AstraZeneca

Alternative Names: AZD-2373; ION-532; IONIS-AZ5-2.5Rx

Latest Information Update: 11 Sep 2025

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At a glance

Originator Ionis Pharmaceuticals
Developer AstraZeneca
Class Antisense oligonucleotides; Urologics
Mechanism of Action Gene silencing

Orphan Drug Status

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

No
New Molecular Entity Yes

Highest Development Phases

Phase II Kidney disorders

Most Recent Events

18 Aug 2025 AstraZeneca initiates phase-I pharmacokinetics and safety trial in USA (SC, injection) (NCT07154901)
28 May 2025 No recent reports of development identified for phase-I development in Kidney-disorders(In volunteers) in USA (SC, Injection)
05 Mar 2025 Phase-II clinical trials in Kidney disorders in USA (SC) (NCT06824987) (AstraZeneca pipeline, June 2025)

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