Purpose

Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported.

Methods

After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.

Results

In children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104. In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104. No new adverse effects of vosoritide were detected.

Conclusion

Vosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years.

中文翻译：

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vosoritide 对软骨发育不全儿童的安全和持久的生长促进作用：开放标签、3 期扩展研究的 2 年结果

 目的

软骨发育不全是由成纤维细胞生长因子受体 3 基因的致病性变异引起的，导致软骨内骨化受损。 Vosoritide 是 C 型钠尿肽的类似物，可刺激软骨内骨生长，目前正在开发用于治疗软骨发育不全的药物。这项 3 期扩展研究旨在记录软骨发育不全儿童连续每日使用 vosoritide 治疗的有效性和安全性，并报告了两年的结果。

 方法

在完成至少六个月的基线观察生长研究和 52 周的双盲安慰剂对照研究后，参与者有资格继续开放标签扩展研究的治疗，其中所有参与者均接受剂量为 vosoritide 的伏索立肽治疗。 15.0微克/公斤/天。

 结果

在随机接受 vosoritide 治疗的儿童中，年化生长速度从基线时的 4.26 厘米/年增加到 52 周时的 5.39 厘米/年和 104 周时的 5.52 厘米/年。速度从第 52 周的 3.81 厘米/年增加到第 104 周的 5.43 厘米/年。未检测到沃索立肽新的不良反应。

 结论

沃索立肽治疗对软骨发育不全儿童具有安全且持久的生长促进作用，每日治疗两年。