Trial Profile

A phase 2/3 trial of BMN 333 in Achondroplasia patients.

Status: Planning

Phase of Trial: Phase II/III

Latest Information Update: 08 Oct 2025

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At a glance

Drugs BMN 333 (Primary)
Indications Achondroplasia
Focus Registrational; Therapeutic Use

Most Recent Events

08 Oct 2025 New trial record
04 Aug 2025 Based on results from Phase 1 healthy volunteers study, BioMarin is advancing plans to initiate the registration-enabling Phase 2/3 study in the first half of 2026.

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