Sanofi pol­ish­es rare dis­ease pro­file as Nie­mann-Pick drug scores promis­ing PhII/III da­ta — there's just one ques­tion

Two years af­ter it en­tered Sanofi’s R&D spot­light, an en­zyme re­place­ment ther­a­py has come through in a piv­otal study — paving the way for reg­u­la­to­ry sub­mis­sions in 2021 af­ter the drug­mak­er an­swers one re­main­ing ques­tion.

Rare dis­ease con­tin­ues to be a stat­ed fo­cus un­der new CEO Paul Hud­son even as he ex­e­cutes a dras­tic shake­up on the pipeline, ex­cis­ing di­a­betes and car­dio re­search while bur­nish­ing on­col­o­gy.

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