Sanofi's Paul Hud­son scores an FDA OK for a rare dis­ease drug — af­ter a lengthy R&D odyssey

Right on the heels of an EMA rec­om­men­da­tion for EU ap­proval, Sanofi has scooped up the FDA’s OK for the first ap­proved ther­a­py to treat symp­toms that are not re­lat­ed to the cen­tral ner­vous sys­tem in pa­tients with acid sphin­gomyeli­nase de­fi­cien­cy, which in­cludes a range of ail­ments un­der the um­brel­la for Nie­mann-Pick dis­ease.

For Sanofi, the ap­proval of the en­zyme re­place­ment ther­a­py Xen­pozyme (Olipu­dase al­fa) marks a key win for Paul Hud­son’s strat­e­gy to beef up their rare dis­ease port­fo­lio, but this one has been a long time com­ing. Sanofi picked up the drug af­ter the 2011 ac­qui­si­tion of Gen­zyme and post­ed pos­i­tive piv­otal da­ta two and a half years ago.

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