Barth syndrome, which is estimated to affect around 150 people in the United States, has no approved treatments. It typically affects boys and causes heart and muscle weakness, as well as delayed growth.
The FDA did not respond to a Reuters request for comment
The latest setback comes after a 16.5-month review, during which the FDA extended its review from January to April to assess additional data. Last month, the regulator missed the extended deadline.
“We don’t understand why it wasn’t an approval, because there isn’t really a request that we resubmit new data,“ said CEO Reenie McCarthy.
In October, a panel of advisers to the FDA voted 10-6 in favor of the therapy.
FDA raised the possibility of an accelerated approval based on the findings, but is hesitant to extend the pathway to critically ill newborns, who make up nearly two-thirds of the therapy’s expanded access program participants.
The company said it will meet with the FDA next month.
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