The big question after Stealth’s PDUFA delay
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Is the PDUFA delay for Stealth BioTherapeutics a canary-in-the-coalmine moment? Or is it an overblown one-off that signals little about disruption at the FDA?
That’s the question seemingly on everyone’s minds after the FDA on Tuesday delayed a PDUFA date for Stealth's new drug application. There weren’t safety or manufacturing concerns, the agency didn’t ask Stealth to conduct additional trials, and Stealth doesn’t know when a decision might get made.
FDA Commissioner Marty Makary has pointed out that decision dates aren’t hard deadlines, but rather “target dates” the agency attempts to meet. That’s technically true: The law mandates that agency reviews meet a minimum of only 90% of the PDUFA dates it sets. “It's not like missing a statutory deadline,” Patricia Zettler, an Ohio State professor who specializes in FDA law, told me this week.
But the delay of Stealth’s PDUFA (as well as a delay for a post-CRL hearing from Vanda Pharmaceuticals) has people asking whether other stages of the regulatory process could be impacted. There are copious amounts of FDA work not tied to deadlines, like biomarker qualification programs, that could be slowed even if the FDA keeps largely hitting PDUFA dates.
So far, most biopharma companies have said — both on earnings calls and in interviews with Endpoints — they aren’t seeing any impact on FDA reviews. And many recent PDUFA dates have been met without issues, including for Dupixent, Amvuttra, Sanofi’s RNAi drug for hemophilia and rare disease products from Amgen and J&J.
Those applications coming through on time provide a significant counterpoint to the Stealth delay, Leerink analyst Daina Graybosch said. While Graybosch believes the agency will be affected by the layoffs (“it’s inevitable,” she said), it’s almost certainly too early to know how the changes at the FDA are going to affect US drug development and regulation. (Makary has argued that the regulator was overstaffed and inefficient before the cuts.)
Graybosch didn’t follow the Stealth case closely, but she noted its exceedingly long development history and its drug having been previously rejected and delayed.
“Blaming this on DOGE, I'm sorry, there's so many things going on and ways and reasons to see risk,” Graybosch said. “Taking what happened at [Stealth] and reading into it more broadly feels alarmist to me.”
But other analysts and FDA experts I spoke to agreed that the Trump administration’s healthcare staffing cuts are going against its stated goal of making government more efficient, and that firing hundreds of staff and slashing review teams will likely make the agency less effective at its job.
“It may be too early to say things are chugging along just fine,” Zettler said. “Bigger questions are, ‘What about products that are earlier in development or are not as far along in their review process?’”
— Max Gelman, senior editor
Here's more of our FDA coverage:
- FDA delays PDUFA date for small biotech, raising questions about impact of agency cuts: The fate of a small, privately-held biotech’s new drug has been delayed, in what appears to be a sign that broad cuts to the FDA by the Trump administration may be affecting key regulatory decisions.
- Exits continue at FDA, as more staff in drug office follow fired colleagues out the door: The FDA office that approves new drugs is seeing more staff leave in early retirements, following broad cuts this month that targeted almost 20% of workers at the agency.