FDA action on Vertex drug based on lab tests

Kimberly Doheny, lab director at the Center for Inherited Disease Research at Johns Hopkins University, holds an example of a slide used for testing DNA , Friday, March 21, 2008, in Baltimore. The DNA scanners used in the lab, blue boxes each about twice as big as a desktop printer, will reveal what DNA "letter" appears in more than 580,000 spots in the genetic material, said Doheny. (AP Photo/Rob Carr)

Vertex Pharmaceuticals scored a minor coup this week when the Food and Drug Administration signed off on a wider use of its banner drug for cystic fibrosis. But the decision wasn’t based on a large, rigorous clinical trial — or any study involving humans at all. Instead, the FDA took the word of a test tube.

The agency looked at lab-test results and determined that Vertex’s drug, Kalydeco, would benefit an additional number of cystic fibrosis patients, pointing out that the drug had already demonstrated its safety in earlier human trials.

It’s an unprecedented decision, and two FDA scientists explained it at length on the agency’s website. But experts say it’s unlikely to signal a loosening of standards at the US drug regulator, and Wall Street analysts questioned whether it’s much of a departure from standard practice in the first place.


“FDA is sending this signal that it’s something they’re willing to consider, which resonates with the broader policy discussion about being more flexible,” said Patti Zettler, a former FDA attorney who is now an associate professor at Georgia State University College of Law. “But clearly it has to be the right situation.”

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According to the FDA, Kalydeco fit the bill because its safety and efficacy in cystic fibrosis is already well understood. And the additional patients now eligible for the drug number just 900, making a proper clinical trial less than feasible, the agency said.

Damian Garde can be reached at Follow him on Twitter @damiangarde.