Drug Profile

BMN 349

Alternative Names: BMN-349

Latest Information Update: 05 Jun 2025

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At a glance

Originator BioMarin Pharmaceutical
Class Small molecules
Mechanism of Action Alpha 1-antitrypsin modulators; Protein folding stabiliser

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 Alpha 1-antitrypsin deficiency

Most Recent Events

15 Nov 2024 Pharmacodynamics data from a preclinical study in alpha-1 antitrypsin deficiency presented at the 75th Annual Meeting of the American Association for the Study of Liver Diseases (AASLD-2024)
04 Sep 2024 BioMarin Pharmaceutical plans proof of concept (POC) study for Alpha-1 antitrypsin deficiency in 2026
05 Aug 2024 BioMarin Pharmaceuticals plans a phase I trial in healthy volunteers by the end of 2024

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