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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER