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TA Base Rate
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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 is not yet 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 focused on rare diseases. BioMarin trades on the Nasdaq under the ticker symbol BMRN.
BMN 333 is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or other regulatory agencies and is still undergoing clinical trials to evaluate its safety and efficacy.
BMN 333 is being studied in a Phase 2 clinical trial with the identifier NCT07441876. This active, recruiting study evaluates the efficacy and safety of BMN 333 compared to vosoritide in children with achondroplasia, with an enrollment target of 160 participants.
No, BMN 333 is not the same as vosoritide. In the ongoing Phase 2 trial, BMN 333 is being compared against vosoritide, which serves as the active control. Both target the CNP pathway but are distinct investigational agents.