BioMarin eyes FDA approval as PhIII results emerge in most common form of dwarfism
BioMarin’s drug for the most common cause of dwarfism appears to be making kids taller. The company announced topline final results from its Phase III trial, showing vosoritide improved annual growth by 1.6 cm against placebo in children between the ages of 5 and 14 with achondroplasia.
The 1.6 cm and its 0.0001 p-value qualifies the trial as a success but falls short of the 2.0 cm analysts had hoped for. Still, with these results and the latest Phase II extension data showing vosoritide increased growth by 9.0 cm over 54 months, BioMarin is planning on pre-submission marketing meetings with health authorities in the first half of 2020.
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