Drug offers hope for dwarfism sufferers
By Hallie Jeon
An experimental drug to treat dwarfism in children is showing positive results.
BioMarin Pharmaceutical has developed the drug for achondroplasia, the most common form of dwarfism.
"In children receiving the highest dose of 15 micrograms per kilogram daily, we observed a 50-percent increase in the mean annualized growth rate compared to their own growth rate," said Wolfgang Dummer, vice president of BioMarin.
"If maintained, this increase in growth could allow children with achondroplasia to resume a normalized growth rate, and we have observed no major safety concerns to date," he said.
In the study, the 10 children who were given the highest dose of the drug grew at an average of 6.1 centimeters a year, which is about a 50-percent increase from their previous growth rate.
The new growth rate was similar to that of a child without achondroplasia, the study said.
Dr. William Wilcox, a human genetics professor at Emory University, urged caution, saying there were only 10 children given the high dose and they were only treated for six months.
"Six-month results are important, but they don't really give you the whole story," he said.
In fact, human growth hormone, a drug already marketed for certain children, might not have lasting effects after one year, he said. It would be important to see if the experimental drug could demonstrate lasting effects.
The drug BMN111, or vosoritide, is expected to be available in North America, Europe, Asia and Latin America when approved.