Back­lash: Har­vard ex­perts fret over the fall­out from FDA’s OK for Sarep­ta’s con­tro­ver­sial Duchenne drug

Two of Har­vard’s top FDA pol­i­cy ex­perts, Aaron Kessel­heim and Jer­ry Avorn, have ex­pressed some deep con­cerns about the agency’s ap­proval of eteplirsen, OK’d to be sold as Ex­ondys 51 for $300,000-plus a year, and the im­pli­ca­tions for fur­ther show­downs in the near fu­ture as more de­vel­op­ers look to test the bound­aries on the agency’s stan­dards.

Writ­ing on­line in the Jour­nal of the Amer­i­can Med­ical As­so­ci­a­tion, the two re­viewed the cam­paign used to back eteplirsen, win­ning an OK with scant ev­i­dence of suc­cess in pro­duc­ing dy­s­trophin — con­trary to what Sarep­ta had claimed — a prob­lem­at­ic re­liance on his­tor­i­cal com­par­isons, and the unan­i­mous op­po­si­tion of the main re­view­ers, in­clud­ing se­nior of­fi­cials who ap­pealed Janet Wood­cock’s lone in­sis­tence on an ac­cel­er­at­ed ap­proval.

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