FDA shoots down a biotech’s last-stand pa­tient pitch af­ter fault­ing da­ta

Stealth Bio­Ther­a­peu­tics ap­peared un­sure its pitch for an ul­tra-rare dis­ease would be ac­cept­ed when sub­mit­ting an NDA in Au­gust, and on Wednes­day its fears came to pass.

The FDA is­sued Stealth a re­fusal to file let­ter for a can­di­date look­ing to treat Barth syn­drome, the biotech an­nounced Wednes­day morn­ing. It’s a move not en­tire­ly un­ex­pect­ed, giv­en Stealth’s own ad­mis­sion ear­li­er this year that the agency didn’t find ex­ist­ing da­ta to sup­port NDA re­view.

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