FDA Lashes Back at Sarepta’s Arguments

It’s been a tough road for Duchenne candidates this year, with one getting rejected outright and another facing an unsympathetic advisory committee panel next last week.

In new documents posted ahead of the meeting, FDA staff appeared unmoved by arguments submitted by Sarepta Therapeutics in support of its Duchenne muscular dystrophy candidate eteplirsen, at points even countering the company’s position.

The agency made it clear that it doesn’t agree with Sarepta’s “characterization of inaccuracies” in briefing documents the FDA released prior to a canceled Jan. 22 meeting, when the candidate was originally scheduled to be reviewed by the Peripheral and Central Nervous System Drugs Advisory Committee. That meeting was postponed due to inclement weather.