For 3rd time, the FDA and its new commissioner dropped safety issues and trial demands, clearing the path for snubbed therapy

TherapeuticsMD $TXMD was slammed on May 8 when the FDA formally rejected its application for a vaginal pain medicine dubbed TX-004HR. Regulators wanted more safety data, which would have required a new study and years more work.

Then, one day later, Scott Gottlieb won confirmation as the new commissioner of the FDA, and that may have made a huge difference in the biotech’s fortunes.

This morning TherapeuticsMD, which had said it would appeal the decision, says the FDA has relinquished its demands and will allow them to resubmit their application in a matter of weeks. Instead of a pre-approval study, regulators say they will now be satisfied with a post-approval trial.

The biotech’s stock rocketed up 116%.

Once rare, these sudden about-faces by the FDA have become almost routine this year, with TherapeuticsMD lining up as one of three companies to win a huge reprieve — signaling a much more tolerant attitude at the FDA in light of President Donald Trump’s mandate to push more drug approvals faster as a way of bringing down pharma costs in the US.

All three had applications rejected before Gottlieb’s arrival.

Robert Finizio

Amicus Therapeutics got this trend started in July — two months after Gottlieb took the head office — when the FDA dropped its demand for a safety study of its Fabry disease drug migalastat, which battered the biotech’s shares. In a follow-up, the FDA put the drug on its fast track program to speed up the new review.

Four months after Gottlieb showed up at his new office at the FDA, Eli Lilly announced that the FDA had reversed course on its blockbuster application for baricitinib, also rejected on safety grounds. As Lilly told me at the time:

The FDA has granted us the opportunity to file our resubmission package without a new clinical study.

TherapeuticsMD makes three, signaling that the agency’s old standards on safety have clearly been downgraded. While the agency has not been reluctant to spurn new drug applications, a new day has dawned on where the agency draws the line on safety signals. And that has major implications for drug developers harried by questions of safety issues.

I asked Gottlieb about the agency’s safety standards and a response to this story, but media contacts at the FDA did not immediately respond.

While Amicus and Eli Lilly both made cases for leading therapies in their pipelines, TherapeuticsMD has been seeking an approval for a me-too drug that will go up against other well established therapies. Regulators had objected to the 12 weeks of safety data provided in the application, saying they wanted 12 months of results. But now they have been overruled.

Another consideration: If any of these drugs now being helped toward the market backfire and hurt patients in unexpected ways, the FDA and Gottlieb will have plenty of explaining to do.

“We appreciate the hard work and collaborative effort by the FDA in moving TX-004HR one step closer to approval,” said TherapeuticsMD CEO Robert Finizio. “We are extremely pleased with the FDA’s position that an additional pre-approval safety study is no longer necessary for the resubmission of the NDA for TX-004HR.”

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

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