FDA shoots down a biotech’s last-stand patient pitch after faulting data

Stealth BioTherapeutics appeared unsure its pitch for an ultra-rare disease would be accepted when submitting an NDA in August, and on Wednesday its fears came to pass.

The FDA issued Stealth a refusal to file letter for a candidate looking to treat Barth syndrome, the biotech announced Wednesday morning. It’s a move not entirely unexpected, given Stealth’s own admission earlier this year that the agency didn’t find existing data to support NDA review.