Phase 2 Trial of Vosoritide Use in Patients with Hypochondroplasia: A Pharmacokinetic/Pharmacodynamic Analysis
Authors' conclusion
Vosoritide treatment showed improvement in AHV and height SD in children with hypochondroplasia. PK analysis indicates that drug exposure was correlated to global CNP activity as measured by urine cGMP but did not correlate with growth outcomes. More studies are needed to identify specific patient characteristics that can predict response to therapy and clinical outcomes.
Study context
- Population
- Prepubertal children with hypochondroplasia (12 females, mean age 5.9 ± 2.3 years, mean height −3.29 + 0.68 SD)
- Participants
- 24 children
- Outcome
- Correlations of PK/PD parameters with growth outcomes
Methodological detailssingle-arm
- Single-arm
Main finding
As reported in the abstract
Max change in cyclic guanosine monophosphate (cGMP) AUC correlated with PK AUC
r = 0.46
p = 0.0001
CXM levels correlated with prior 6-month interval height velocity (partial correlation coefficient)
0.40
p = 0.0048
Further findings
Serum CXM · baseline to 12 months
- CXM increased from baseline mean 22.5 ± 6.5 to 41.6 ± 15.9 ng/mL (p < 0.0001)
Urine cGMP
- Urine cGMP increased within 1 h and peaked at 2 h after injection
- Mean cGMP AUC not significantly different at each study visit
Vosoritide PK parameters
- PK parameters similar to those previously reported in achondroplasia
Original publication
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