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BioMarin's dwarfism drug increases children's growth in phase 3 trial

BioMarin Pharmaceutical Inc.'s experimental treatment for a form of genetic short stature increased children's rate of height growth, meeting the main goal of a late-stage study.

The phase 3 study for vosoritide, which is intended to treat the most common form of disproportionate short stature called achondroplasia, enrolled 121 children aged five to 14. This age range was selected because the children were still growing. The once-daily injection was evaluated against placebo for a year.

Children taking 15 micrograms per kilogram of vosoritide daily saw a 1.6 centimeter per year growth velocity compared to those taking placebo, BioMarin said in a Dec. 16 press release. Secondary goals in the trial were body and limb proportionality, bone growth and health, functional independence and quality of life. These endpoints are still being evaluated in an extended study treating all enrolled participants.

BioMarin said vosoritide was generally well-tolerated and did not cause clinically significant decreases in blood pressure. The most common adverse event was injection site reactions, which subsided over time.

San Rafael, Calif.-based BioMarin now intends to meet with regulatory authorities in early 2020 to discuss a potential application for drug approval.

Achondroplasia, which occurs in approximately one in 25,000 live births worldwide, is caused by a mutation in a bone growth gene called the fibroblast growth factor receptor 3 gene, or FGFR3. Achondroplasia can lead to health complications such as sleep apnea, recurrent ear infections, bowed legs and incomplete development. Available treatments include invasive surgeries to decompress the spinal cord or straighten bowed legs.

Short stature caused by a genetic or medical condition is medically referred to as dwarfism.