FDA hurdles put drug used to treat rare disorder in limbo

A treatment for Barth syndrome, a rare and often fatal genetic disorder, faces unexpected FDA-related hurdles, NBC News reported Aug. 18. 

Approximately 150 people in the U.S. have Barth syndrome, a condition that can cause heart failure. Some with the disorder die before their 5th birthday; two infant deaths were reported recently, according to NBC News. 

Some have benefited from an experimental drug called elamipretide. However, the drug has not been approved for full market use, and its manufacturer, Stealth BioTherapeutics, recently was required to resubmit a new drug application. 

After more than a decade of development, elamipretide appeared to be close to receiving approval. An FDA advisory committee voted in favor of the medication in October, citing how it improved heart function and muscle strength in clinical trials. But in May, the FDA rejected the drug without explanation. 

Rep. Buddy Carter of Georgia has called on the FDA for clarity, saying elamipretide is “really the only drug that we know of that works,” NBC News reported.

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