FDA opens up an eth­i­cal can of worms with ac­cel­er­at­ed ap­proval for first drug for un­der­ly­ing ge­net­ic cause of dwarfism

The FDA on Fri­day signed off on an ac­cel­er­at­ed ap­proval for Bio­Marin’s Vox­zo­go (vosori­tide) in­jec­tion, the first treat­ment to tar­get the un­der­ly­ing ge­net­ics of dwarfism, which can in­crease the height of chil­dren five years of age and old­er with the con­di­tion.

The in­jec­tion, which Bio­marin said will have an an­nu­al net price of $240,000, works by bind­ing to a spe­cif­ic re­cep­tor called the na­tri­uret­ic pep­tide re­cep­tor-B, which re­duces the growth reg­u­la­tion gene’s ac­tiv­i­ty and stim­u­lates bone growth in chil­dren.

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