Home›Trial›NCT03424018
NCT03424018Phase 3ACTIVE NOT RECRUITING

An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

Study Summary

The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia

Conditions
Achondroplasia
Interventions / Drugs
BMN 111
View on ClinicalTrials.gov ↗
Sponsor
BMRN
BioMarin Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment119 participants
Primary Completion—
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531295+00:00
What This Trial Means for Biotech Investors
Trial Phase Context

Phase 3 trials are pivotal studies with large patient populations (hundreds to thousands) designed to prove efficacy and safety for FDA submission. A successful Phase 3 readout is typically the last major hurdle before a New Drug Application (NDA) or BLA filing.

Status & Expected Data

Enrollment is complete. The trial is in its follow-up or data collection phase — a readout may occur on or near the primary completion date.

Enrolled: 119 participants
Sponsor Catalyst Profile

BioMarin Pharmaceutical (BMRN) is the sponsoring company for this trial. BiotechSign currently grades this company D (49/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.

BMRN Full Pipeline →All Companies →
Not financial advice. BiotechSign data is sourced from ClinicalTrials.gov, SEC EDGAR, and other public databases for research and informational purposes only. It does not constitute investment recommendations. Always do your own due diligence before making investment decisions.