Stealth BioTherapeutics’ New Drug Application for elamipretide, a therapy for Barth syndrome, received an expedited FDA review target date of September 26, significantly earlier than the anticipated six-month standard timeline. This decision follows multiple FDA rejections and substantial public and congressional advocacy underscoring the urgency for this treatment of a severe ultra-rare disease. The accelerated review offers hope for patients facing limited options, though the sponsor reports financial strain and staff reductions linked to regulatory delays.