Stealth BioTherapeutics Receives Orphan Drug Designation from FDA for Elamipretide for Treatment of Duchenne Muscular Dystrophy

Pre-IND meeting request granted by FDA’s Division of Neurology I

Excerpt from the Press Release:

BOSTON, May 12, 2022 /PRNewswire/ — Stealth BioTherapeutics Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today that the US Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation to elamipretide for the treatment of patients with Duchenne muscular dystrophy (DMD). 

The company also announced that the FDA’s Division of Neurology I has granted its request for a pre-IND meeting to discuss a development path for elamipretide in combination with products within the approved therapeutic class of exon-skipping phosphorodiamidate morpholino oligomers (PMO).  The company has previously presented data demonstrating that administration of elamipretide in combination with a PMO significantly improves dystrophin expression levels in the X-linked muscular dystrophy (mdx) mouse model.  The company also previously conducted a pre-IND meeting with the FDA’s Division of Cardiology and Nephrology during which it aligned on a potential development path for elamipretide for cardiomyopathy associated with DMD.

“We are pleased that the FDA has recognized the high unmet need for innovative treatments for DMD,” said Chief Executive Officer Reenie McCarthy. “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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