Bio­Marin sub­mits NDA for con­tro­ver­sial achon­dropla­sia drug; Cell and gene ther­a­py boom sparks €49M buy­out

Bio­Marin is plow­ing ahead with its achon­dropla­sia can­di­date, sub­mit­ting an NDA to the FDA for vosori­tide Thurs­day af­ter­noon.

If ap­proved, the drug would be a once-dai­ly in­jec­tion for chil­dren with the con­di­tion, which is the most com­mon cause of dwarfism. The ap­pli­ca­tion fol­lows the re­lease of topline Phase III tri­al re­sults back in De­cem­ber show­ing vosori­tide im­proved an­nu­al growth by 1.6 cm against place­bo in chil­dren be­tween the ages of 5 and 14.

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