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Vosoritide

Phase 3

Hypochondroplasia | Small molecule | Other |BioMarin Pharmaceutical Inc.|Last Updated: Mar 31, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment280
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaPHASE3 ENROLLING BY_INVITATION 140 — — Jun 20, 2025Dec 1, 2040Jul 18, 202523 United States, Australia +7
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaPHASE3 ACTIVE NOT_RECRUITING 80 — — Jun 17, 2024Aug 1, 2026Jan 20, 202623 United States, Australia +7
NCT07126262A Study of Vosoritide Versus Placebo in Children With Hypochondroplasia Aged 0 to < 36 MonthsPHASE2 RECRUITING 60 — — Jul 30, 2025Jun 30, 2028Mar 31, 202626 United States, Australia +5
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Study Endpoints
Primary Endpoints
Evaluate the long-term efficacy of vosoritide treatment until final adult height (FAH)
Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males

As measured by change from baseline in height Z-score

Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
At week 52
Incidence of treatment-emergent adverse events
From baseline to end of treatment at 52 weeks
Incidence of serious adverse events versus placebo over the course of the study
From baseline to end of treatment at 52 weeks
Changes from baseline in standard clinical laboratory values (hematology, urinalysis, and chemistry)
At week 26, at week 52
Changes from baseline in heart rate
At week 13, at week 26, at week 39, at week 52

Units of measure: bpm

Change from baseline in height Z-score
At week 52
Changes from baseline in respiratory rate
At week 13, at week 26, at week 39, at week 52

Units of measure: breaths/min

Changes from baseline in temperature
At week 13, at week 26, at week 39, at week 52

Units of measure: celsius

Changes from baseline in blood pressure
At week 13, at week 26, at week 39, at week 52

Units of measure: mmHg

Secondary Endpoints
Evaluate the maintenance effect of vosoritide treatment on standing height
Baseline to week 52
Evaluate the maintenance effect of vosoritide treatment on standing height Z-score
Baseline to week 52
Evaluate long-term effect of vosoritide treatment on growth velocity
Annually from enrollment through Final Adult Height, defined as 16 years of age for females and 18 years of age for males
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
VosoritideEXPERIMENTALOpen-label active drug
vosoritide injection with vial and syringeEXPERIMENTAL -
Placebo injection with vial and syringePLACEBO_COMPARATOR -
Interventions
NameTypeDescription
VosoritideDRUGOpen-label administration of vosoritide using weight-band dosing
PlaceboDRUGSubcutaneous injection of recommended dose of placebo
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Eligibility Criteria
Age Range3 Years — 17 Years
SexALL
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: * Participants must have completed the Week 52 visit for 111-303 or 111-212 and have open epiphyses as assessed by left hand antero-posterior (AP) X-rays * Parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study...

Countries:United StatesAustraliaCanadaFranceGermanyItalyJapanSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT07126262primaryCompletionDate: changed
LOWMay 26, 2026NCT07073014primaryCompletionDate: changed
LOWMay 26, 2026NCT06455059primaryCompletionDate: changed
LOWMay 24, 2026NCT07126262studyFirstPostDate: changed
LOWMay 24, 2026NCT07073014studyFirstPostDate: changed
LOWMay 24, 2026NCT06455059studyFirstPostDate: changed