Using Vosoritide to Treat Hypochondroplasia by Inhibiting the Fibroblast Growth Factor Receptor 3 Signaling Pathway
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Reviewed by Dat Tien Nguyen, B.A, ScM.
Translated by Nhi Phuong Quynh Le, B.A |
Posted on May 24th, 2024
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Hypochondroplasia is hallmarked by the over-activation of the fibroblast growth factor receptor 3 (FGFR3) which disrupts endochondral ossification. Vosoritide is an analogue of c-type natriuretic peptide that can bind to and inhibit the mitogen-activated protein kinase pathway downstream from FGFR3 activation. A study recently published on the Lancet had investigated the effect of Vosoritide on treating hypochondroplasia.
The phase 2 clinical trial included 24 children between the age of 3 and 11 who had not entered puberty. The baseline height of these children was shorter than 99.9% of the children with the corresponding age in the United States. The average growth velocity of the cohort was 5.12 cm/year which was less than 87.3% of the children of the same age. Due to the rare nature of the condition, there were not many participants in the study; thus, the researchers did not include a control group. All of the participants received a daily subcutaneous injection containing Vosoritide at a dose of 15 micrograms per kilogram of body weight. After 12 months of treatment, the researchers observed that Vosoritide significantly increased the annualized growth velocity to 6.93 cm/year, and the participants’ height increased to reach the median level of children of the corresponding age. Due to the small sample size, the researchers could not investigate the likelihood of rare adverse events.
The phase 2 clinical trial included 24 children between the age of 3 and 11 who had not entered puberty. The baseline height of these children was shorter than 99.9% of the children with the corresponding age in the United States. The average growth velocity of the cohort was 5.12 cm/year which was less than 87.3% of the children of the same age. Due to the rare nature of the condition, there were not many participants in the study; thus, the researchers did not include a control group. All of the participants received a daily subcutaneous injection containing Vosoritide at a dose of 15 micrograms per kilogram of body weight. After 12 months of treatment, the researchers observed that Vosoritide significantly increased the annualized growth velocity to 6.93 cm/year, and the participants’ height increased to reach the median level of children of the corresponding age. Due to the small sample size, the researchers could not investigate the likelihood of rare adverse events.