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news.Stealth BioTherapeutics has received Orphan Drug Designation from the US Food and Drug Administration (FDA) Office of Orphan Products Development for elamipretide to treat Duchenne muscular dystrophy (DMD) patients.
DMD is a fatal disease of muscle membrane instability. Credit: Dr. Edwin P. Ewing, Jr. / commons.wikimedia.org/
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Additionally, the FDA’s Division of Neurology I granted its request for a pre-IND meeting for discussing a development path for elamipretide along with products within the approved exon-skipping phosphorodiamidate morpholino oligomers (PMO) therapeutic class.
Earlier, Stealth BioTherapeutics submitted data which showed that elamipretide administration in combination with a PMO significantly improves levels of dystrophin expression in the X-linked muscular dystrophy (mdx) mouse model.
Furthermore, the company carried out a pre-IND meeting with the Division of Cardiology and Nephrology of the FDA during which it aligned on a potential development path for the therapy for cardiomyopathy linked to DMD.
Stealth BioTherapeutics CEO Reenie McCarthy said: “We are pleased that the FDA has recognised 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.”
The X chromosome-linked genetic disorder DMD is caused by mutations in the DMD gene that encodes the dystrophin protein.
Typically diagnosed in childhood, DMD is a fatal disease of muscle membrane instability and is associated with progressive skeletal muscle dysfunction.
Upon receiving Orphan Drug Designation, an investigational drug gets various development benefits including exclusivity for seven years on obtaining marketing approval.
The company is also evaluating its second-generation clinical-stage candidate, SBT-272, for rare neurological disease indications.
