FDA gives Merck ‘real-time’ OK for Keytruda, offering a peek at the latest extension of the agency’s data express highway

The FDA doesn’t always wait for a marketing application to come in before it begins its review these days. And that suits Merck just fine.

The federal agency has stamped a full approval — following last year’s conditional OK — on Merck’s mega-blockbuster Keytruda for frontline use in a chemo combo for fighting non-squamous non-small cell lung cancer. Regulators based their decision on the data set from KEYNOTE-189, put on display last April, one in a series of late-stage trials that Merck has been rolling out to grab the lead on the PD-1/L1 market from a group of tough competitors at Bristol-Myers Squibb.

The green light is anything but unexpected. But it is noteworthy for reasons that the entire industry should be paying attention to.

Roger Perlmutter

What distinguishes this particular OK is the timing. FDA commissioner Scott Gottlieb has been pushing a new pilot program called real-time oncology reviews, where regulators start their work ahead of a formal application. The first real-time OK went to Novartis a few weeks ago for two combinations using Kisqali against breast cancer. And top-tier drug developers are finding that instead of just being greeted with an open door, sometimes the door is being taken off the hinges entirely.

In this case they were dealing with a hazard ratio of 0.49, marking a 51% drop in the risk of death for patients taking the combo. There’s still no median survival data for the combo available, but the matchup of the Keytruda/chemo approach versus chemo alone provided a clear set of advantages for Merck.

  • The median progression-free survival rate was 8.8 months for the combo, 4.9 months for chemo alone.
  • The overall response rate was 48% compared to 19% in the control.
  • Median response duration hit 11.2 months for the Keytruda arm and 7.8 months for control.

These real-time reviews now on offer at the FDA look to extend the data express highway that’s been built for the agency’s breakthrough drug designation and other hurry-up campaigns that have transformed the speed and design of oncology studies over the past few years. In this case the FDA wants to rely entirely on US studies, excluding anything outside the borders, while preferring drugs that are delivering clear benefits and are being tested on some obvious endpoints. And they don’t want any formulation changes to ponder when making a snap call.

The FDA is taking no chances with Keytruda, a landmark drug by any definition that is being pushed through hundreds of combination studies and new approaches. Gottlieb is likely to earn even more devotion from the industry’s leaders for following through on improving the FDA’s responsiveness. And Merck is happy to have the fresh set of bragging rights today.

“Keytruda is rapidly becoming a foundation for the treatment of appropriate patients with metastatic non-small cell lung cancer,” said Merck R&D chief Roger Perlmutter. “Today’s approval of the expanded label for Keytruda based on data from the KEYNOTE-189 trial is an important milestone.”


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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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