FDA grants orphan drug designation for Duchenne muscular dystrophy treatment

Stealth BioTherapeutics Corp. announced that the FDA has granted orphan drug designation to elamipretide for the treatment of Duchenne muscular dystrophy.

The company also announced in a press release that the FDA’s division of neurology has granted a meeting request to discuss a development path for elamipretide in combination with products within the approved therapeutic class of exon-skipping phosphorodiamidate morpholino oligomers.

“We are pleased that the FDA has recognized the high unmet need for innovative treatments for [Duchenne muscular dystrophy],” Stealth CEO Reenie McCarthy said in the release. “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.”

Duchenne muscular dystrophy (DMD) is an X chromosome-linked genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. DMD is a disease of muscle membrane instability, where the loss of dystrophin and mitochondrial dysfunction results in progressive muscle loss.

Typically diagnosed in children, DMD is initially associated with skeletal muscle dysfunction, which progresses to loss of ambulation in early adolescence and eventual assisted ventilation in early adulthood.