Eu­ro­pean reg­u­la­tors are lock­ing the door on Sarep­ta’s Duchenne drug — but they aren’t throw­ing away the key

Sarep­ta Ther­a­peu­tics $SRPT won’t be sell­ing its Duchenne mus­cu­lar dy­s­tro­phy drug eteplirsen in Eu­rope any­time soon.

Eu­ro­pean reg­u­la­tors at the CHMP con­firmed their thumbs-down on the drug, Sarep­ta CEO Doug In­gram said, re­sist­ing the biotech’s at­tempt to change their po­si­tion and set­ting up an all-but-cer­tain for­mal re­jec­tion by the EMA.

Eteplirsen — sold in the US as Ex­ondys51 — re­mains a high­ly con­tro­ver­sial drug. Ap­proved al­most sole­ly on the in­sis­tence of Janet Wood­cock, who over­rode ob­jec­tions from the re­view team and se­nior FDA of­fi­cials, the drug’s la­bel in­cludes the note that the com­pa­ny has yet to prove its ef­fi­ca­cy.

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