Sarep­ta rock­ets up af­ter FDA ap­proves its con­tro­ver­sial Duchenne drug

Af­ter years of fierce de­bate in a see­saw bat­tle over the fate of Sarep­ta’s Duchenne mus­cu­lar dy­s­tro­phy drug eteplirsen, the FDA has grant­ed the drug con­di­tion­al ap­proval in the US, mak­ing this the first drug OK’d to treat the lethal dis­ease.

The ap­proval comes with the threat of swift ret­ri­bu­tion, though, if in­ves­ti­ga­tors fail to fol­low through with pos­i­tive da­ta from a big­ger study. The agency’s state­ment in­cludes a re­quire­ment that Sarep­ta will now have to con­firm that the drug ac­tu­al­ly works as de­signed, spurring pro­duc­tion of dy­s­trophin in a way that can im­prove mo­tor func­tion in the 13% of pa­tients amenable to ex­on 51 skip­ping. And the agency says that it may with­draw its OK with­out that da­ta.

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