FDA to expedite review of rare disorder treatment

The FDA has accepted Stealth BioTherapeutics’ resubmission of its new drug application for elamipretide, a treatment for Barth syndrome.  The agency set an earlier review target date of Sept. 26, ahead of the Feb. 15 deadline, according to an Aug. 21 news release from the drugmaker.  Approximately 150 people in the U.S. have Barth syndrome, a rare disorder that can cause heart failure. This move comes after decades of development and the FDA rejecting the drug in May with no explanation. 

The company had resubmitted the application Aug. 15 after receiving a complete response in May, and the submission seeks accelerated approval based on data showing more than 45% improvement in muscle strength in a phase 2 trial. A postmarketing trial is also planned to confirm clinical benefits, the rele