Rare dis­ease drug­mak­er files for ap­proval de­spite ob­jec­tions from FDA on ad­e­quate tri­al da­ta

An­oth­er small biotech at­tack­ing a su­per-rare dis­ease is lever­ag­ing a pa­tient com­mu­ni­ty and very lim­it­ed clin­i­cal da­ta to try to win an FDA ap­proval.

Stealth Bio­Ther­a­peu­tics, a Cam­bridge biotech that has trad­ed as a pen­ny stock since its lead drug failed a Phase II tri­al in 2019, on Tues­day an­nounced plans to sub­mit a new drug ap­pli­ca­tion to the FDA for that same lead drug for a dif­fer­ent dis­ease: The ul­tra-rare Barth syn­drome, a po­ten­tial­ly fa­tal ge­net­ic dis­or­der that can weak­en heart mus­cles over time.

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