Chiesi, Pro­tal­ix's Fab­ry dis­ease treat­ment is ap­proved as Elfab­rio joins Fab­razyme, Galafold

The FDA has green­lit an en­zyme re­place­ment ther­a­py for adults with a rare, in­her­it­ed dis­ease in which ab­nor­mal de­posits of fat­ty sub­stances build up and cause pain and some­times end-or­gan fail­ure — two years af­ter a CRL and just days af­ter Eu­rope ap­proved the ther­a­py.

The ap­proval for Chiesi Glob­al Rare Dis­eases and Pro­tal­ix Bio­Ther­a­peu­tics’ Elfab­rio puts the treat­ment in the same are­na as Sanofi’s Fab­razyme and Am­i­cus Ther­a­peu­tics’ Galafold in treat­ing Fab­ry dis­ease. Sanofi’s ther­a­py was giv­en an ac­cel­er­at­ed ap­proval in the ear­ly aughts, but a full ap­proval didn’t come un­til 2021. Am­i­cus al­so orig­i­nal­ly re­ceived an FDA re­jec­tion, though it wound up se­cur­ing a nod in 2018.

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