Stealth BioTherapeutics announced via news release that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to elamipretide, a mitochondrially targeted tetrapeptide, for the treatment of Duchenne muscular dystrophy (DMD).

In addition, they announced that their request for a pre-Investigational New Drug (IND) meeting on the development of elamipretide with the FDA’s Division of Neurology has been granted.

“We are pleased that the FDA has recognized the high unmet need for innovative treatments for DMD,” Reenie McCarthy, chief executive officer of Stealth BioTherapeutics said. “We look forward to further discussions with the FDA regarding our development initiatives, which we hope will bring new options to patients suffering from this devastating disease.”

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Stealth BioTherapeutics believes elamipretide could treat DMD and other mitochondrial disorders based on research demonstrating that the mitochondrial dysfunction associated with DMD limits the ability of cells to incorporate or retain exon-skipping phosphorodiamidate morpholino oligomers (PMOs) and PMO-mediated dystrophin.

Previously, the company presented data showing that combining elamipretide with PMOs significantly improved expression levels of dystrophin in an animal model of X-linked muscular dystrophy, and they have already conducted a pre-IND meeting with the FDA’s Division of Cardiology and Nephrology on a possible development path for elamipretide for DMD-associated cardiomyopathy.


Stealth BioTherapeutics receives Orphan Drug designation from FDA for elamipretide for treatment of Duchenne muscular dystrophy. News release. Stealth BioTherapeutics; May 12, 2022.