Stealth BioTherapeutics Corp (NASDAQ:MITO) received Orphan Drug Designation to Elamipretide from the United States Food and Drug Administration (FDA) for the treatment of patients with Duchenne muscular dystrophy (DMD).
Reenie McCarthy, Chief Executive Officer, said, ”We are pleased that the FDA has recognized the high unmet need for innovative treatments for DMD, 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.”
Stealth Bio's Pre-IND meeting request was granted by FDA's Division of Neurology I, to discuss a development path for elamipretide in combination with products within the approved therapeutic class of exon-skipping phosphorodiamidate morpholino oligomers (PMO).
Duchenne muscular dystrophy (DMD) is a fatal disease of muscle membrane instability in which the loss of dystrophin and ensuing mitochondrial dysfunction results in a cascade of events leading to progressive loss of muscle function.
FDA grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. It also provides benefits of seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.
Stealth BioTherapeutics shares are trading high 47 percent at $0.28 in the pre-market session.
