EC ap­proves Bio­Marin drug as first treat­ment for chil­dren with com­mon form of dwarfism; Servi­er sN­DA wins ap­proval

The Eu­ro­pean Com­mis­sion just ap­proved its first drug for use in chil­dren with achon­dropla­sia, a com­mon form of dwarfism.

Bio­Marin re­ceived mar­ket­ing au­tho­riza­tion to­day for Vox­zo­go, al­so known as vosori­tide. The drug is a once-dai­ly in­jec­tion to treat achon­dropla­sia in chil­dren from the age of 2 un­til growth plates are closed, which oc­curs af­ter pu­ber­ty.

An es­ti­mat­ed 11,000 chil­dren across Eu­rope, Mid­dle East, and Africa are af­fect­ed by achon­dropla­sia — and a third of this pop­u­la­tion are in coun­tries au­tho­rized un­der the EMA li­cense. The French Na­tion­al Agency for Med­i­cines and Health Prod­ucts Safe­ty al­so grant­ed Vox­zo­go a “Tem­po­rary Au­tho­riza­tion for Use” to al­low im­me­di­ate ac­cess.

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