Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Label indication VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity [see Clinical Studies (14) ] . Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). FDA label
Also known as Vosoritide Injection, BMN 111, Vosoritide Injection [Voxzogo], Modified recombinant human C-type natriuretic peptide
Vosoritide · 13 trials · 4 indications
As measured by change from baseline in height Z-score
Long term efficacy as measured by change in annualized growth velocity
AGV at a Post-baseline Visit is defined as \[(Height at Post-baseline Visit - Height at Baseline)/(Date of Post-baseline Visit - Date of Baseline Assessment)\] x 365.25 AGV at Baseline is defined as \[(Height at Baseline - last height measurement in Study 111-901 at least 6 months prior to Baseline)/(Date of Baseline Assessment - Date of last height measurement in Study 111-901 at least 6 months prior to Baseline)\] x 365.25
Units of measure: bpm
Units of measure: breaths/min
Units of measure: celsius
Units of measure: mmHg
Number of study participants with treatment-emergent adverse events or serious adverse events
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. A severity grade was defined by the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03. As per CTCAE, Grade 1 scales as Mild; Grade 2 scales as Moderate; Grade 3 scales as severe or medically significant but not immediately life threatening; Grade 4 scales as life-threatening consequences; and Grade 5 scales as death related to AE. Safety Population includes all sentinel and randomized participants in the FAS who received at least one dose of vosoritide or placebo in this study. Serious adverse event (SAE)
Z-Scores were derived using age-sex specific reference data (means and SDS) for average stature children per the Centers for Disease Control and Prevention. A height Z score of 0 would indicate that the subject's height is equal to the mean height for the average stature population of the same sex and age. A positive height Z score indicates that the subjects height is above the mean height for the average stature population of the same sex and age, whilst a negative height Z score indicates that the subjects height is below the mean height for the average stature population of the same sex and age. To conclude if the height Z score increases then this means the height deficit has decreased. standard deviation score (SDS). The primary efficacy analysis population was the subset of randomized participants in the FAS.
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. Serious adverse event (SAE).
A treatment-emergent Adverse Events (TEAE) is any Adverse Events that newly appeared, increased in frequency or worsened in severity following initiation of study drug administration. TEAE - Treatment-emergent adverse event. SAE - Serious adverse event.
| Arm | Type | Description |
|---|---|---|
| Vosoritide | EXPERIMENTAL | Open-label active drug |
| vosoritide injection with vial and syringe | EXPERIMENTAL | - |
| Placebo injection with vial and syringe | PLACEBO_COMPARATOR | - |
| BMN 111 | EXPERIMENTAL | - |
| Active BMN 111 | EXPERIMENTAL | Daily subcutaneous injection of 15 micrograms per kilogram BMN111 |
| Placebo | PLACEBO_COMPARATOR | Daily subcutaneous injection of placebo |
| Vosoritide Dose 1 - Low Dose | EXPERIMENTAL | Drug: Vosoritide - Dose 1 Injection • Experimental Drug Lyophilized powder for reconstitution |
| Vosoritide Dose 2 - Medium Dose | EXPERIMENTAL | Drug: Vosoritide - Dose 2 Injection • Experimental Drug Lyophilized powder for reconstitution |
| Vosoritide Dose 3- High Dose | EXPERIMENTAL | Drug: Vosoritide Dose 3 Injection • Experimental Drug Lyophilized powder for reconstitution |
| Vosoritide Dose 3 - High Dose | EXPERIMENTAL | Vosoritide Dose 3 daily injection |
| Human Growth Hormone | ACTIVE_COMPARATOR | hGH daily injection (US only) |
| Vosoritide + Standard of Care | EXPERIMENTAL | Standard of Care treatment for cervicomedullary compression and once daily subcutaneous injection of recommended dose of vosoritide based on weight-band dosing. |
| Standard of Care Alone | NO_INTERVENTION | Institutional standard of care monitoring and treatment for cervicomedullary compression |
| Active BMN111 | EXPERIMENTAL | Subcutaneous injection of 15 μg/kg/day and/or 30 μg/kg/day of BMN111 daily. |
| BMN 111 - Subcutaneous Injection | EXPERIMENTAL | 111-205 is an open-label, extension study. Subjects receive the same stable dose of BMN 111 received upon completion of the 111-202 study, initially up to 30 μg/kg. BMN 111 will be administered by weight-band dosing regimen. |
| Cohort 1 | EXPERIMENTAL | Cohort 1: 2.5 ug/kg |
| Cohort 2 | EXPERIMENTAL | Cohort 2: 7.5 ug/kg, |
| Cohort 3 | EXPERIMENTAL | Cohort 3: 15 ug/Kg |
| Cohort 4 | EXPERIMENTAL | Cohort 4: 30 ug/kg |
| Name | Type | Description |
|---|---|---|
| Vosoritide | DRUG | Open-label administration of vosoritide using weight-band dosing |
| Placebo | DRUG | Subcutaneous injection of recommended dose of placebo |
| BMN 111 | DRUG | Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. |
| Vosoritide Injection | DRUG | Modified recombinant human C-type natriuretic peptide Vosoritide |
| Human Growth Hormone | DRUG | Commercial product containing somatotropin |
| Active BMN 111: Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. | DRUG | Modified recombinant human C-type natriuretic peptide (subject to adjustment per protocol) |
| Normal Saline | DRUG | SC injection, Part 1 single dose and Part 2 multiple dose |
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...
Vosoritide is an investigational small molecule developed by BioMarin Pharmaceutical Inc. for growth-related rare diseases. It is being studied in achondroplasia, hypochondroplasia, idiopathic short stature, and Noonan syndrome. It is also known as BMN 111 and is marketed under the name Voxzogo.
Vosoritide is being developed to treat achondroplasia, hypochondroplasia, idiopathic short stature, and Noonan syndrome. These are rare growth disorders affecting children. Clinical development includes trials in hypochondroplasia and Noonan syndrome, with enrollment across multiple countries including the United States, Europe, and Japan.
Vosoritide targets NPR2, the natriuretic peptide receptor 2. It is classified as a binding agent that acts on this receptor. NPR2 signaling plays a role in bone growth regulation, which is the biological pathway relevant to the growth disorders vosoritide is being studied in.
Vosoritide is developed by BioMarin Pharmaceutical Inc., which trades under the ticker BMRN. BioMarin is the sponsor of the clinical trials evaluating vosoritide in achondroplasia, hypochondroplasia, idiopathic short stature, and Noonan syndrome.
Vosoritide is in Phase 3 clinical development. It is an investigational therapy and has not been established as approved for the indications currently under study. The program includes both Phase 2 and Phase 3 trials across hypochondroplasia and Noonan syndrome, with 8 total trials and 4 currently active.
Vosoritide trials include NCT07126262, a Phase 2 study versus placebo in children with hypochondroplasia aged 0 to under 36 months, and NCT07073014, a Phase 3 long-term extension study in hypochondroplasia. NCT06455059 is a Phase 3 hypochondroplasia study, and NCT06668805 is a Phase 2 study in Noonan syndrome.
Yes, vosoritide is also known as BMN 111. Other names include vosoritide injection and the brand name Voxzogo. The subcutaneous formulation is described as a once-daily weight-band based dosing regimen. These names all refer to the same BioMarin Pharmaceutical development program.