FDA agrees to review a rare disease drug that its developer was about to give up on

File this under “Not a moment too soon.”

After years of back-and-forth deliberations, the U.S. Food and Drug Administration has agreed to review a rare disease drug developed by a small company that planned to shelve further work if the agency did not take this long-sought step.

Specifically, the FDA accepted a new drug application from Stealth BioTherapeutics for a treatment to combat Barth syndrome, which causes an enlarged heart, muscle weakness, and a shortened life expectancy. The agency also announced it would hold an advisory committee meeting to review the application, but no date has been set. However, the FDA did not issue a priority review, which could delay approval.