Stealth BioTherapeutics Submits Elamipretide New Drug Application to FDA for Treatment of Barth Syndrome
"We initiated our Barth syndrome development efforts at the request of the Barth syndrome community.
- "We initiated our Barth syndrome development efforts at the request of the Barth syndrome community.
- Stealth believes, however, that the data could support an NDA review, and has accordingly submitted the NDA as requested by the Barth syndrome patient community.
- Elamipretide was previously granted rare pediatric designation, fast track designation, and orphan drug designation by the FDA, and orphan drug designation by the EMA, for the treatment of Barth Syndrome.
- Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide.