Stealth BioTherapeutics has resubmitted its regulatory application for an investigational therapy targeting Barth syndrome, an ultra-rare genetic disorder, following an FDA rejection earlier in the year. The company has publicly disclosed the FDA’s rejection letter as part of efforts to rally support for the drug’s approval. Stealth faces financial and operational pressures pending FDA review outcomes. The therapy is currently available to a limited group of patients under an expanded access program, with advocates emphasizing its life-saving potential. The company is cautiously optimistic about a forthcoming FDA decision.