FDA rejects TherapeuticsMD, citing lack of long-term data

FDA's rejection of TherapeuticsMD’s TX-004HR could significantly delay approval

The FDA has rejected a filing for approval of TherapeuticsMD’s treatment for vaginal pain during sexual intercourse because of the lack of 12-month safety data. The news leaves TherapeuticsMD looking down the barrel of an expensive, time-consuming safety study for TX-004HR unless it can persuade FDA to accept another route to approval.

TherapeuticsMD filed for, and expected to win, approval of the estradiol vaginal softgel capsule on the strength of a 12-week study. That expectation was shattered by a complete response letter, in which TherapeuticsMD says the FDA informed it long-term endometrial safety data are needed for approval. TherapeuticsMD thinks those data are the only thing standing between it and approval. But for a company with $113.5 million in cash targeting an increasingly-competitive niche, the time and cost of the safety study make it a significant barrier to approval and commercial success.

Management is hoping to persuade the FDA it can allay the safety concerns without running a study and has requested a Type A meeting to make its case. Such meetings take place within 30 days of the request, meaning TherapeuticsMD should no one way or another whether it can pull off the Hail Mary pass and win round FDA soon.

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TherapeuticsMD is going into the meeting with a long list of grievances.

“There are numerous reasons why we disagree with the FDA’s response. One, throughout the TX-004HR clinical program we had numerous discussions with the FDA regarding the safety and efficacy design of the trial to support approval. The FDA did not once tell us to perform a 12-month endometrial safety study or tell us that our 12-week trial design was inadequate to support approval,” TherapeuticsMD CEO Robert Finizio said on a conference call with investors.

Finizio went on to give more detail on the meetings and written correspondence TherapeuticsMD had with the FDA, before launching into a rundown of five other reasons he disagrees with the rejection. Those additional points covered the FDA guidance on products such as TX-004HR and the precedent set by approvals of similar drugs.

If these arguments fail to persuade the FDA to approve TX-004HR without a 12-month safety study, TherapeuticsMD faces a significant delay in bringing the drug to market given such trials are unavoidably time consuming. But Finizio, in the belief there are viable alternative paths to approval, is unwilling to say just how big a blow this eventuality would be for TherapeuticsMD.

“I don't want to speculate on the time and length for an additional trial because we don’t know if that’s necessary. There are probably a number of different paths forward. Once we know there is a trial needed—and that’s not said in the complete response, it’s the lack of long-term data—then we’ll certainly bring it forward,” he said.

Investors were more willing to interpret the likely harm caused to TherapeuticsMD by the FDA knockback. Shares in TherapeuticsMD fell 10% following the release of the news, driving its market cap down to $883 million.