Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
BioMarin · Achondroplasia
Phase 2 / Phase 3
Recruiting
160
2026-07
Industry sponsor intelligence
BioMarinClinical Pipeline.
Lead-sponsor view of BioMarin's indexed peptide studies, development phases, active programs, completed work and recent registry changes.
- Total
- 15
- Recruiting
- 4
- Active
- 6
- Completed
- 4
- Results posted
- 3
- Avg enrollment
- 77
Pipeline overview
Programs by phase and activity.
Velocity
BioMarin trials by start year
Study start year · indexed snapshot
Development
Trials by phase
16phase labels
Early Phase 10
Phase 12
Phase 29
Phase 35
Phase 40
| Product | Studies | Early / I | Phase II | Phase III | Phase IV | Active |
|---|---|---|---|---|---|---|
| Vosoritide → | 15 | 2 | 9 | 5 | 0 | 10 |
Active peptide trials
In motion now.
NCT IDStudyPhase / statusEnrollmentUpdated
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
BioMarin · Idiopathic Short Stature
Phase 2
Recruiting
100
2026-05
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
BioMarin · Noonan Syndrome
Phase 2
Recruiting
30
2026-05
A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 Months
BioMarin · Hypochondroplasia
Phase 2
Recruiting
60
2026-03
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
30
2026-03
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
BioMarin · Achondroplasia
Phase 3
Active, not recruiting
119
2026-03
An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
73
2026-03
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
BioMarin · Achondroplasia
Phase 2
Active, not recruiting
20
2026-03
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
BioMarin · Hypochondroplasia
Phase 3
Active, not recruiting
80
2026-01
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
BioMarin · Hypochondroplasia
Phase 3
Enrolling by invitation
140
2025-07
Completed trials
Finished registry records.
NCT IDStudyPhase / statusEnrollmentUpdated
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
BioMarin · Achondroplasia
Phase 2
Completed
75
2024-06
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
BioMarin · Achondroplasia
Phase 3
Completed
121
2022-03
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
BioMarin · Achondroplasia
Phase 2
Completed
35
2021-01
A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult Volunteers
BioMarin · Achondroplasia
Phase 1
Completed
74
2012-06
Search sponsor records
All BioMarin studies.
This page groups studies where BioMarin is listed as the lead industry sponsor. Collaborator-only records are not included in the count.
15 matching studies
Last registry update first
Recent updates
Latest registry movement.
NCT07441876Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With AchondroplasiaJul 24, 2026NCT06382155A Phase 2 Study of Vosoritide in Children With Idiopathic Short StatureMay 22, 2026NCT06668805A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentMay 11, 2026NCT07126262A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 MonthsMar 31, 2026NCT02724228A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)Mar 13, 2026NCT03424018An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaMar 13, 2026NCT03989947An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaMar 13, 2026NCT04554940A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaMar 13, 2026NCT06455059Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaJan 20, 2026NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaJul 18, 2025