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Vosoritide

Phase 3

Hypochondroplasia | Small molecule | Rare Disease |BioMarin Pharmaceutical Inc.|Last Updated: Aug 24, 2026

Target and mechanism

Molecular targetNPR2
Target classBinding Agent
ModalitySmall molecule
ChEMBLCHEMBL3707276

Also known as Vosoritide Injection, Vosoritide Injection [Voxzogo]

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment280

FDA Designations

No designations recorded

Clinical trial landscape

Vosoritide · 6 trials · 4 indications

Phase 3 2Phase 2 4
NCT07073014Long-Term Extension Study of Vosoritide to Treat Children With HypochondroplasiaHypochondroplasia
ENROLLING BY_INVITATION140 Analytics
NCT06455059Interventional Study of Vosoritide for the Treatment of Children With HypochondroplasiaHypochondroplasia
ACTIVE NOT_RECRUITING80 Analytics
PHASE3ENROLLING BY_INVITATION
Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
Hypochondroplasia
NCT07073014140
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PHASE3ACTIVE NOT_RECRUITING
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
Hypochondroplasia
NCT0645505980
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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

Change from baseline in Annualized Growth Velocity (AGV)
At 6 months
Change from baseline in height
At 4 years
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Through Week 260

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 -
Vosoritide Dose 1 - Low DoseEXPERIMENTALDrug: Vosoritide - Dose 1 Injection • Experimental Drug Lyophilized powder for reconstitution
Vosoritide Dose 2 - Medium DoseEXPERIMENTALDrug: Vosoritide - Dose 2 Injection • Experimental Drug Lyophilized powder for reconstitution
Vosoritide Dose 3- High DoseEXPERIMENTALDrug: Vosoritide Dose 3 Injection • Experimental Drug Lyophilized powder for reconstitution
Vosoritide Dose 3 - High DoseEXPERIMENTALVosoritide Dose 3 daily injection
PlaceboPLACEBO_COMPARATORPlacebo daily injection
Human Growth HormoneACTIVE_COMPARATORhGH daily injection (US only)
Vosoritide + Standard of CareEXPERIMENTALStandard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of recommended dose of vosoritide based on weight-band dosing.
Standard of Care AloneNO_INTERVENTIONInstitutional standard of care monitoring and treatment for cervicomedullary compression

Interventions

NameTypeDescription
VosoritideDRUGOpen-label administration of vosoritide using weight-band dosing
PlaceboDRUGSubcutaneous injection of recommended dose of placebo
Vosoritide InjectionDRUGModified recombinant human C-type natriuretic peptide Vosoritide
Human Growth HormoneDRUGCommercial product containing somatotropin
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Eligibility Criteria

Age Range3 Years to 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 KingdomSouth Korea
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Recent Changes (Last 90 Days)

LOWAug 24, 2026NCT06668805lastUpdatePostDate: changed
LOWAug 24, 2026NCT06668805lastUpdatePostDate: changed

Frequently asked questions about Vosoritide

What is Vosoritide used for?

Vosoritide is an investigational therapy being studied for achondroplasia, hypochondroplasia, Noonan syndrome, and idiopathic short stature. It is a small molecule peptide developed by BioMarin Pharmaceutical Inc. (BMRN). Vosoritide is currently in Phase 3 clinical development for these growth-related conditions.

What does Vosoritide target?

Vosoritide is a peptide that belongs to the -tide class of molecules. It is being studied for its effects on growth in children with achondroplasia, hypochondroplasia, Noonan syndrome, and idiopathic short stature. The specific molecular target of Vosoritide is not disclosed in the available clinical trial information.

Who makes Vosoritide?

Vosoritide is developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company listed on NASDAQ under the ticker BMRN. BioMarin is conducting clinical trials to evaluate Vosoritide for achondroplasia, hypochondroplasia, Noonan syndrome, and idiopathic short stature.

What phase is Vosoritide in?

Vosoritide is in Phase 3 clinical development. It is being evaluated in a Phase 3 trial for hypochondroplasia (NCT06455059) and in Phase 2 trials for achondroplasia, idiopathic short stature, and Noonan syndrome. Vosoritide is investigational and has not been approved by regulatory authorities.

What clinical trials is Vosoritide in?

Vosoritide is being studied in four clinical trials. NCT04554940 evaluates safety in infants with achondroplasia. NCT06382155 studies children with idiopathic short stature. NCT06455059 is a Phase 3 trial for hypochondroplasia. NCT06668805 examines children with Noonan syndrome. All trials are active, with a total planned enrollment of 280 participants.

Is Vosoritide the same as Voxzogo?

Vosoritide is also known as Vosoritide Injection and Vosoritide Injection [Voxzogo]. The name Voxzogo appears in the alternative names for Vosoritide, indicating that Voxzogo is a brand name associated with this drug. Vosoritide is being developed by BioMarin Pharmaceutical Inc.