The FDA and the EMA go their sep­a­rate ways on DMD: CHMP slaps down Sarep­ta but of­fers ri­val PTC an ex­pand­ed mar­ket

Eu­ro­pean and US reg­u­la­tors oc­ca­sion­al­ly take com­plete­ly con­flict­ing stands on drugs, and nowhere is that more ev­i­dent than in Duchenne mus­cu­lar dy­s­tro­phy.

To­day, Eu­rope’s CHMP turned thumbs down on Sarep­ta’s $SRPT Ex­ondys51 (eteplirsen) two years af­ter the FDA ap­proved it for mar­ket­ing in one of the most con­tro­ver­sial de­ci­sions in the agency’s his­to­ry. And the same group em­braced an ex­pan­sion of PTC’s $PTC ataluren, even though the drug has failed re­peat­ed clin­i­cal tri­als and has been snubbed now sev­er­al times by the FDA for its un­proven ef­fi­ca­cy pro­file.

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