FDA Sets September 26 Review for Stealth’s Barth Syndrome Drug; Soleno Faces 415-Page Safety Report and Recall Petition

The U.S. Food and Drug Administration (FDA) has agreed to expedite the review of Stealth BioTherapeutics' drug elamipretide, intended to treat Barth syndrome, an ultrarare disease. The agency accepted the company's third new drug application and set a target decision date of September 26, 2025. This follows an initial rejection of the drug in May 2025 after its original submission in January 2024. Separately, Soleno Therapeutics is responding to a 415-page short-seller report alleging safety concerns with its newly approved therapy VYKAT XR, used for treating Prader-Willi syndrome. Soleno's CEO, Anish Bhatnagar, disputed claims from the report by Kir Kahlon that patients are experiencing severe adverse effects, emphasizing the importance of post-marketing safety surveillance. Meanwhile, the short-seller group Scorpion Fund filed a citizen petition with the FDA requesting a voluntary recall of VYKAT XR, pending confirmation from the agency. Additionally, Capricor Therapeutics is preparing to challenge the FDA's rejection of its therapy for Duchenne muscular dystrophy (DMD) cardiomyopathy, with former FDA official Prasad returning to the agency to assist in the effort.