Rare disease drug specialist BioMarin (BMRN +1.02%) recently shared successful top-line results from a clinical trial of vosoritide, an injected treatment intended to increase growth rates for children born with achondroplasia. On Saturday, the company will present results from the pivotal study at this year's meeting of the American Society for Bone and Mineral Research (ASMBR) in a virtual oral presentation to take place at 11:50 a.m. EDT.
Achondroplasia occurs in between 1 in 15,000 and 1 in 40,000 newborns. This makes the hereditary condition the most common form of short-limbed dwarfism, and there are currently no effective treatment options for it on the market. Those born with achondroplasia have trouble converting cartilage into bone, particularly in the arms and legs. While shorter stature is the most obvious symptom, complications of the condition can also include life-threatening spine malformation and chronic pain.
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During the trial, children treated with daily injections of vosoritide grew significantly faster than those receiving a placebo. At the 52 week check-in, the average growth velocity of children given vosoritide was 0.62 inches per year faster than the placebo group.
The European Medicines Agency began reviewing a marketing application for vosoritide on Aug. 13, and BioMarin submitted a new drug application to the FDA on Aug. 20. The FDA has until Oct. 19 to request more information from BioMarin or begin its review process, which generally takes 10 months to complete.
