Stealth Bio gets FDA ad­comm for pre­vi­ous­ly re­ject­ed ul­tra-rare dis­ease drug

Sev­en years af­ter Stealth Bio­Ther­a­peu­tics land­ed a fast track des­ig­na­tion from the FDA, the agency has fi­nal­ly agreed to re­view the com­pa­ny’s ap­pli­ca­tion for an ul­tra-rare, ge­net­ic Barth syn­drome treat­ment.

Known as elamipre­tide, the drug will face an FDA ad­vi­so­ry com­mit­tee. The ap­pli­ca­tion won’t get a pri­or­i­ty or ac­cel­er­at­ed re­view from the agency, the Mass­a­chu­setts-based com­pa­ny said Mon­day.

The ad­comm is like­ly to fea­ture a tough dis­cus­sion and vote be­cause there are no oth­er ap­proved treat­ments for the fa­tal dis­ease, and Stealth is sub­mit­ting da­ta from an open-la­bel ex­ten­sion com­pared to a ret­ro­spec­tive nat­ur­al his­to­ry study, and ad­di­tion­al sup­port­ing ef­fi­ca­cy and safe­ty da­ta from a nat­ur­al his­to­ry study.

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