Vosoritide
CNP analogue
Vosoritide é um análogo de CNP avaliado em ensaios clínicos de acondroplasia, incluindo um estudo fase 3 randomizado e controlado por placebo.[1]
01 / Overview
Quick facts
Somente campos atualmente documentados aparecem neste registro.
- class
- CNP analogue
- molecule Type
- Peptide analogue
- primary Target
- Growth plate chondrocytes
- receptor
- NPR-B
- primary Axis
- CNP / cGMP
- research Status
- Approved drug
- developer
- BioMarin
What is it studied for?
Evaluated in a 52-week phase 3 randomized, double-blind, placebo-controlled trial.
02 / Mechanism
03 / Evidence synthesis
What does the evidence actually show?
As categorias evitam misturar evidência humana, dados pré-clínicos e plausibilidade mecanística. A cobertura desta versão não é uma revisão sistemática.
- The phase 3 trial found a greater change in annualized growth velocity at 52 weeks than placebo.
Data not indexed yet.
- The trial did not establish final adult height or long-term harms.
Data not indexed yet.
Data not indexed yet.
Claims review not indexed yet.
04 / Protocolos publicados
Doses relatadas em estudos humanos publicados
Os valores reproduzem protocolos descritos em pesquisas publicadas. Eles não constituem recomendação de uso, prescrição ou orientação clínica.
| Estudo | População | N | Via | Dose relatada | Frequência | Duração | Desenho | Desfecho primário | Referência |
|---|---|---|---|---|---|---|---|---|---|
| Ravi Savarirayan et al. | Ambulatory children aged 5 to <18 years with achondroplasia | 121 | Subcutaneous | 15.0 µg/kg | Once daily | 52 weeks | human-rct · not-extracted | Change from baseline in annualized growth velocity at 52 weeks | Abrir |
06 / Safety
Safety & Adverse Events
Reported findings
- Adverse events were common in both study groups; no serious adverse event was considered treatment-related in the trial report.
Pharmacological concerns
Data not indexed yet. Lack of indexed harm data is not evidence of safety.
Unknowns
- Final adult height and long-term harms were not established by the 52-week study.
07 / Source literature
Studies
Multiple entry points
Related Paths
Vosoritide can also be explored through these curated graph routes.
No dead ends
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Growth Plate
topic
Cartilagem organizada responsável pelo crescimento longitudinal ósseo durante o desenvolvimento.
Chondrocytes
topic
Células especializadas da cartilagem e da placa de crescimento.
CNP / NPR-B Signaling
pathway
Sinalização do receptor guanilato ciclase NPR-B por CNP, relevante à biologia da cartilagem de crescimento.
Bone
tissue
Mineralized connective tissue maintained by coordinated formation and resorption.
Cartilage
tissue
Avascular connective tissue whose specialized cells maintain a proteoglycan-rich matrix.
Growth Plate
tissue
Organized cartilage zones that support longitudinal bone growth before epiphyseal closure.
NPR2 / NPR-B
target
NPR2 / NPR-B is a membrane guanylyl cyclase receptor connected to cGMP, PKG, RAF / MEK / ERK modulation.
Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial
study
Provides phase 3 evidence for increased annualized growth velocity in the studied pediatric achondroplasia population.
09 / References
References
- [1]
Ravi Savarirayan, Louise Tofts, Melita Irving, William Wilcox, Carlos A. Bacino, et al.. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. The Lancet. 2020.