Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 111 · 6 trials · 1 indication
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
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.
* Number of study participants with treatment-emergent adverse events. * Number of study participants with treatment-emergent 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. 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 |
|---|---|---|
| BMN 111 | EXPERIMENTAL | - |
| Active BMN 111 | EXPERIMENTAL | Daily subcutaneous injection of 15 micrograms per kilogram BMN111 |
| Placebo | PLACEBO_COMPARATOR | Daily subcutaneous injection of placebo |
| 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 |
|---|---|---|
| BMN 111 | DRUG | Subcutaneous injection of recommended dose of BMN 111 based on weight-band dosing once daily. |
| Placebo | DRUG | Subcutaneous injection of 15 μg/kg of placebo daily |
| Normal Saline | DRUG | SC injection, Part 1 single dose and Part 2 multiple dose |
Inclusion Criteria: * Must have completed Study 111-301 * Female \>= 10 years old or who have begun menses must have a negative pregnancy test at the Baseline Visit and be willing to have additional pregnancy tests during the study * If sexually active, willing to use a highly effective method of c...
BMN 111 is an investigational small molecule being developed for achondroplasia, a rare genetic disorder of bone growth. It is currently in Phase 3 clinical development and is not yet approved by regulatory authorities.
BMN 111 is being developed by BioMarin Pharmaceutical Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol BMRN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in achondroplasia.
BMN 111 is currently in Phase 3 clinical development for achondroplasia. It is an investigational drug, meaning it has not been approved by regulatory agencies and is still undergoing clinical trials to assess its safety and effectiveness.
BMN 111 has been studied in multiple clinical trials, including NCT01590446 (Phase 1, completed), NCT02724228 (Phase 2, active), NCT03424018 (Phase 3, active), and NCT03583697 (Phase 2, completed). These trials evaluate the drug in healthy volunteers and children with achondroplasia.
BMN 111 is also known as vosoritide, a C-type natriuretic peptide analog being developed for achondroplasia. The drug is designed to promote bone growth by targeting the underlying genetic cause of the condition.