Vosoritide increases bone growth velocity in children with achondroplasia, the most common form of dwarfism, according to a study recently published in The Lancet.
Ravi Savarirayan, M.D., from the University of Melbourne in Australia, and colleagues randomly assigned 121 children diagnosed with achondroplasia (aged 5 to 18 years) to either subcutaneous injections of vosoritide or placebo for 52 weeks.
The researchers found that the adjusted mean difference in annualized growth velocity between patients in the vosoritide group and placebo group was 1.57 cm/year, favoring vosoritide. Most patients (119 people) had at least one adverse event (98 percent in both groups). None of the serious adverse events were thought to be treatment-related, and no deaths occurred.
“It is not known whether final adult height will be increased, or what the harms of long-term therapy might be,” the authors write.
Several authors disclosed financial ties to pharmaceutical companies, including BioMarin, which manufactures vosoritide and funded the study.