For data in a related indication to serve as confirmatory evidence, the study would have to be completed, reviewed by the FDA and determined to demonstrate benefit, OND’s Peter Stein said.
Stein’s comments provide more insight into the FDA’s views of confirmatory evidence under a September 2023 draft guidance.
Elamipretide’s failure in a 12-week endpoint in a Barth syndrome randomized trial informed the design of a Phase III study in primary mitochondrial myopathy, which is running for one year.

Confirmatory Evidence: Related Indication Must Have A Completed Study That Established Benefit, US FDA Says

More from US Advisory Committees