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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