Drug Profile

BMN 351

Alternative Names: BMN-351

Latest Information Update: 26 Feb 2026

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At a glance

Originator BioMarin Pharmaceutical
Class Antisense 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.

No
New Molecular Entity Yes

Highest Development Phases

Phase I/II Duchenne muscular dystrophy

Most Recent Events

24 Feb 2026 Interim pharmacodynamics data from a phase I/II trial in Duchenne muscular dystrophy released by BioMarin Pharmaceutical
04 Sep 2024 BioMarin Pharmaceutical plans proof of concept (POC) study for Duchenne muscular dystrophy in 2025
03 Jan 2024 Phase-I/II clinical trials in Duchenne muscular dystrophy (In children) in Turkey, Netherlands, Italy (IV) (NCT06280209)

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