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NCT07073014Phase 3ENROLLING BY INVITATION

Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia

Study Summary

The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH

Conditions
Hypochondroplasia
Interventions / Drugs
Vosoritide
View on ClinicalTrials.gov ↗
Sponsor
BMRN
BioMarin Pharmaceutical
Rare Disease
D49/100
Study Details
Enrollment140 participants
Primary Completion—
Lead SponsorBioMarin Pharmaceutical
Data Retrieved2026-04-11 02:55:23.531355+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

Current status: ENROLLING BY INVITATION.

Enrolled: 140 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 →
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