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Can Dwarfism Be Treated? BioMarin Pharmaceuticals Thinks So

SAN RAFAEL (CBS SF) -- A San Rafael pharmaceutical company is on its way to helping children with dwarfism grow taller.

BioMarin Pharmaceuticals Inc. scientists announced Wednesday new data that they say show a "durable and consistent effects" from a drug that helps children with dwarfism grow taller.

The new drug, vosoritide, aims to treat the most common form of dwarfism, known as achondroplasia.

According to the World FactBook 2014 edition, the worldwide incidence rate of achondroplasia is about one in 25,000 live births, or about 96,000 potential patients. In Europe, the U.S., Latin America and the Middle East, there are no licensed medications for the condition.

BioMarin states that 80 percent of children with achondroplasia have parents of average stature and that the condition resulted from a spontaneous gene mutation.

BioMarin is planning a phase 3 study to start on children between 5 and 14 years old at the end of 2016.

In the phase 2 study, scientists found that over a one-year period, patients saw 46 percent more growth, or about 1.9 cm.

In the earlier, phase 1 study, BioMarin scientists found that during a six month trial, patients saw a 50 percent increase in their growth, or about 2 cm per year. Other patients saw an increase of 65 percent or about 2.3 cm per year growth.

The company is also planning a separate phase 2 study to evaluate the effect of vosoritide in infants and toddlers.

BioMarin said all adverse events assessed as related to the drug were "mild to moderate" but did include injection site reactions and asymptomatic decreases in blood pressure.

"We're encouraged by the consistency of the data from six to 12 months in both safety and efficacy, and plan to initiate a Phase 3 study by the end of the year," Hank Fuchs, MD, Chief Medical Officer at BioMarin said in a statement Wednesday.  "By addressing the root cause of achondroplasia with vosoritide treatment and normalizing annualized growth velocity in children with achondroplasia, we ultimately hope to improve the medical complications of disproportionate bone growth."

Ravi Savarirayan, M.D., Ph.D., a pediatrics professor at the University of Melbourne and the lead investigator for the BioMarin's vosoritide phase 2 study, states that finding a treatment for achondroplasia has the "potential to decrease medical complications [associated with disproportionate bone growth] and to increase function and quality of life" for patients.

BioMarin notes that a longer treatment period and/or a controlled study may be required to prove that the drug has a positive effect on complications associated with achondroplasia, other than disproportionate short stature.
By Hannah Albarazi - Follow her on Twitter: @hannahalbarazi.

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