BioMarin announces final positive results from the Global Phase III study of vosoritide's treatment of cartilage dysplasia
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Last Update: 2020-06-07
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Source: Internet
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Author: User
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recently, BioMarin(http://published the final positive results of the global Phase III study of Vosoritide (Vsolitide, BMN111) for the treatment of patients with cartilage dysplasia (achondroplasia)About Vosoritide
Vosoritide is a research-based, once-daily c-sodium peptide (CNP) analogue, and cartilage dysplasia is the most common disproportionate lysineThe study, a randomized, double-blind, placebo-controlled Phase III study, involved 121 children aged 5-14 who were still open on the growth board and assessed the efficacy and safety of vosortide tide and placeboThese patients completed at least six months of baseline studies to determine their respective baseline growth rates before entering Phase IIIIn the Phase III study, patients were randomly assigned to a 52-week vosoritide (15ug/kg/day) or placebo treatmentThe main endpoint was a change in the relative baseline growth rate of children treated with vosoritide over a year compared to a placeboThe study reached its main endpoint: one year after treatment, the growth rate of vosoritide treatment was 1.6 cm/year (p 0.0001) relative to the baseline, with a placebo-corrected correctionThe results were consistent with the results in the broad erstofy patient population studiedIn the study, Vosoritide's general tolerance was good, with no clinically significant blood pressure dropIn mid-November, BioMarin published the results of an ongoing, open label, dose discovery phase II study, which showed that patients who received 15?g/kg/day dose of ortidei were significantly higher in cumulative height over a 54-month period (0.00 p.0The data further illustrate the beneficial effects of continued treatment on height in vosoritide, which has increased by 2.2 cm in the past 12 months
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