BioMarin Pharmaceutical Inc. announced one June 14, 2018that the company dosed the first participant in a global Phase 2 study for vosoritide, an analog of C-type Natriuretic Peptide (CNP), in infants and young children with achondroplasia.
このフェーズ2試験は、プラセボ投与群を含む約70名の乳幼児（0-60か月の月齢）を対象に、52週の投与期間を設定します。主目的は、安全性、忍容性、伸長への効果（Z-score；当値が、当群の平均値から、標準偏差 (SD) の乖離を踏まえ算出）を評価することです。
詳細は、プレスリリース参照
“The Phase 2 study is placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia ages zero to less than 60 months for 52 weeks. “
“Children in this study will have completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. “
Refer to the Press release