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Synonyms: BMN 111 | BMN-111 | Voxzogo®
vosoritide is an approved drug (EMA & FDA (2021))
Compound class: Peptide or derivative
Comment: Vosoritide is a cyclic analogue of C-type natriuretic peptide (CNP) that has a longer half-life (duration of action) than the endogenous peptide . It comprises the 37 C-terminal amino acids from human CNP with the addition of proline and glycine (PG) at its N-terminal.
Vosoritide represents a novel mechanism which has the potential to treat mutant fibroblast growth factor receptor 3-driven achondroplasia, by stimulating endochondral ossification and thereby ameliorating impaired bone growth [1-3].
|No information available.|
|Summary of Clinical Use|
|Vosoritide (a.k.a. research code BMN 111) was progressed to Phase 3 clinical evaluation in pediatric patients with achondroplasia. Click here to link to ClinicalTrials.gov's full list of BMN 111 trials. It is administered by once-daily subcutaneous injection.
Vosoritide was granted orphan drug status by both the US FDA and the EMA for the treatment of children with achondroplasia. The EMA approved vosoritide in August 2021, for the treatment of achondroplasia.
|Mechanism Of Action and Pharmacodynamic Effects|
|CNP reduces the effects of over-active FGFR3 that is responsible for the reduced chondrocyte activity and bone growth in achondroplasia . Vosoritide is a CNP mimic that has an extended half-life compared to the endogenous peptide .|
For extended ADME data see the following:
European Medicines Agency (EMA)