Drug Profile

WVE N531

Alternative Names: WVE-N531

Latest Information Update: 23 May 2025

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At a glance

Originator WaVe life Sciences
Class Antisense oligonucleotides; Oligonucleotides
Mechanism of Action Dystrophin expression stimulants; RNA interference

Orphan Drug Status

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

Yes - Duchenne muscular dystrophy
New Molecular Entity Yes

Highest Development Phases

Phase II Duchenne muscular dystrophy

Most Recent Events

08 May 2025 Wave Life Sciences announces intention to submit NDA to US FDA forDuchenne muscular dystrophy in 2026
08 May 2025 Efficacy data from a phase II FORWARD-53 trial in Duchenne muscular dystrophy released by WaVe life Sciences
12 Nov 2024 Wave Life Sciences anticipates regulatory feedback on an accelerated approval pathway in the first quarter of 2025

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