The pow­er of prece­dent: How a one-off de­ci­sion by Janet Wood­cock es­tab­lished an un­in­tend­ed fast path­way at the FDA — what will fol­low?

When CDER chief Janet Wood­cock over­ruled a group of se­nior reg­u­la­tors at the FDA a lit­tle more than 3 years ago to al­low Sarep­ta to start mar­ket­ing eteplirsen for Duchenne mus­cu­lar dy­s­tro­phy, she re­as­sured some of her col­leagues at the agency that it was the on­ly time the biotech would win an ap­proval based on a slight el­e­va­tion of dy­s­trophin pro­duc­tion — a so-called “sur­ro­gate end­point” used to help guide their ac­cel­er­at­ed ap­proval.

The next time Sarep­ta ar­rived with a Duchenne drug — she said in sev­er­al meet­ings about eteplirsen at the FDA — the biotech would be re­quired to prof­fer da­ta from a clin­i­cal tri­al with a hard end­point, ac­cord­ing to a reg­u­la­to­ry ex­pert with a close un­der­stand­ing of the in­ter­nal work­ings of the agency. The biotech would need to prove ef­fi­ca­cy in some­thing like the 6-minute walk test to de­ter­mine if their sec­ond drug, golodirsen, was ac­tu­al­ly help­ing the boys who suf­fered from the dis­ease.

Test­ing an un­proven the­o­ry against the re­al­i­ty of the de­clin­ing strength these boys typ­i­cal­ly face in con­fronting a rare, lethal dis­ease, Wood­cock told her col­leagues, a new ap­pli­ca­tion with hard da­ta in it would give her a chance to de­ter­mine if eteplirsen was pro­vid­ing a safe ben­e­fit to DMD pa­tients and fam­i­lies, and if the slight boost in dy­s­trophin is an ac­cu­rate mark­er for the ben­e­fit.

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