Source:

Press Release

Disclosures: McCarthy is employed by Stealth BioTherapeutics Corp.
May 12, 2022
1 min read
Save

FDA grants orphan drug designation for Duchenne muscular dystrophy treatment

Source:

Press Release

Disclosures: McCarthy is employed by Stealth BioTherapeutics Corp.
You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

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.

Source: Adobe Stock.
Source: Adobe Stock.

“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.