Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
BMN 333 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Phase 2: Low Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: Medium Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: High Dose | EXPERIMENTAL | Participants will be randomized to receive different dose levels of BMN 333 |
| Phase 2: Vosoritide | ACTIVE_COMPARATOR | weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide |
| Phase 3: BMN 333 at selected dose after Phase 2 | EXPERIMENTAL | - |
| Phase 3: Vosoritide | ACTIVE_COMPARATOR | weight band dosing, Modified recombinant human C-type natriuretic peptide Vosoritide |
| Name | Type | Description |
|---|---|---|
| BMN 333 | DRUG | Administration: Weekly subcutaneous injection |
| Vosoritide Injection [Voxzogo] | DRUG | Administration: Daily subcutaneous injection |
Inclusion Criteria: 1. Participants must be aged ≥ 2 to \< 11 years (Phase 2) or ≥ 2 to \< 18 years (Phase 3), at the time of signing the informed consent 2. Participants must have ACH (confirmed by documented genetic testing) and open epiphyses 3. Are Tanner Stage I (Phase 2) or any Tanner stage (...
BMN 333 is an investigational small molecule being developed for achondroplasia, a rare genetic disorder affecting bone growth. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
BMN 333 targets C-type Natriuretic Peptide (CNP), a peptide involved in bone growth regulation. By modulating this pathway, the drug aims to address the underlying growth deficiency in achondroplasia.
BMN 333 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.
BMN 333 is in Phase 2 clinical development. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies and is still being studied in clinical trials.
BMN 333 is being evaluated in a Phase 2 trial with the identifier NCT07441876. This randomized, active-controlled study compares BMN 333 to vosoritide in children with achondroplasia, with an enrollment of 160 participants across multiple countries.
No, BMN 333 is not the same as vosoritide. BMN 333 is a small molecule targeting C-type Natriuretic Peptide (CNP), while vosoritide is a separate treatment for achondroplasia. The two are being compared directly in a Phase 2 clinical trial.