T Time: Women Were First to Put Testosterone Back on FDA’s Agenda. Now It’s Industry’s Turn

More than two decades after FDA last publicly grappled with the development of testosterone for women, the Agency is asking industry to take another look.

On September 17, 2026, FDA’s Office of Women’s Health (OWH) and the Center for Drug Evaluation and Research (CDER) convened a public workshop on testosterone use in menopausal women. The day-long meeting brought together clinicians, researchers, patients, and FDA officials to discuss what is known, what remains unknown, and, importantly for drug developers, what it may take to develop an FDA-approved testosterone product for women.

But perhaps the most remarkable part of the meeting was why it happened at all: women asked for it.

The roots of the workshop can be traced, at least in part, to FDA’s December 2025 public meeting on testosterone replacement therapy in men. More than 80% of comments submitted to the public docket following that meeting came from women, many calling attention to the lack of an FDA-approved testosterone product for women. At last week’s workshop, FDA repeatedly acknowledged the role those voices played in bringing the issue to the forefront.

This is notable in an area that has seen relatively little public regulatory attention for more than 20 years. In 2004, FDA’s Reproductive Health Drugs Advisory Committee considered Procter & Gamble’s Intrinsa, a transdermal testosterone patch developed for hypoactive sexual desire disorder (HSDD) in surgically menopausal women receiving estrogen. Today, in 2026, an FDA-approved testosterone product specifically indicated for women still does not exist.

But the absence of an approved product has not meant the absence of use. Women have been using testosterone products approved for men off-label as well as compounded testosterone formulations for years. The regulatory gap therefore has real world consequences: women are already using testosterone, but without a product developed, studied, approved and labeled specifically for them.

That reality gave the workshop a palatable sense of urgency. FDA was not discussing how to introduce an entirely new therapy into clinical practice. It was asking what evidence should be needed to support an FDA-approved product in an area where use has moved ahead of the regulatory framework.

Why Development Is So Difficult

The meeting also made clear why that regulatory gap has persisted. Testosterone presents several difficult and interconnected development questions, many of which remain unresolved.

The first is deceptively simple: what is the indication? FDA discussed potential areas of investigation including sexual dysfunction, musculoskeletal health, cognition, and mood. But the broad bucket of “testosterone for menopause” is not an indication. Each condition presents different questions about the appropriate population, evidence of clinical benefit, endpoints, dose, and benefit-risk assessment. FDA was clear that the drug and indication go together and that each potential use would need to stand on its own evidence.

Then comes the challenge of demonstrating efficacy. Sexual function trials can have substantial placebo responses, making it difficult to distinguish a treatment effect even when patients improve during a study. That problem is not merely theoretical. During the workshop, panelists discussed the experience with BioSante’s testosterone gel development program, which ultimately failed on efficacy amid a high placebo response and challenges with the sexual function efficacy measure used in the trials.

Even measuring testosterone presents challenges. FDA noted that the “normal” testosterone range in women is poorly defined and discussed variability in assays and measurement of free versus total testosterone. During the regulatory panel, FDA recommended measuring both free and total testosterone, at least initially, and discussed the importance of using sufficiently sensitive and accurate methods, including liquid chromatography-mass spectrometry (LC-MS/MS). While it might seem that at least this challenge would have been conquered in studies of testosterone use in men, an assay that performs adequately at testosterone concentrations typically observed in men may not provide the same performance at the lower concentrations encountered in women. FDA encouraged sponsors to discuss assay strategy with the Agency early to avoid measurement problems later in development.

Given the lack of a well-defined “normal” range of testosterone in women, it is not clear what or even if a particular serum testosterone concentration should serve as the therapeutic target. Individual response may vary, the relationship between circulating testosterone and clinical benefit remains uncertain, and FDA raised the basic question of “how high is too high.” The Agency therefore focused considerable attention on dose finding and exposure-response. Phase 2 may be particularly important for characterizing pharmacokinetics, pharmacodynamics, safety, and clinical response and selecting a dose with a reasonable likelihood of succeeding in Phase 3. The lowest effective dose may also differ depending on the indication.

Combination therapy adds another wrinkle. Women may use testosterone alongside estrogen, with or without a progestogen. FDA discussed the implications of developing testosterone as monotherapy versus as part of a combination regimen. A sponsor developing a fixed combination would need to study the combination and demonstrate the contribution of its individual components to efficacy and safety. Those choices can shape the clinical development program from the outset.

Long-term safety remains perhaps the most consequential evidentiary gap. Existing studies vary in formulation, dose, duration, population, and achieved testosterone exposure. Many randomized trials have been relatively short, while women with higher baseline cardiovascular risk have often been excluded. Questions also remain about breast and endometrial safety. FDA posited that these limitations make it difficult to characterize risks associated with chronic use in the population likely to receive an approved product.

Taken together, the development challenge is formidable. Sponsors must define a clinically meaningful indication and population, select endpoints capable of detecting benefit against what may be a substantial placebo response, establish reliable methods for measuring testosterone concentrations, characterize exposure-response, justify the dose, determine an appropriate treatment regimen, and generate a safety database adequate to support the proposed use.

FDA Is Inviting Conversation

Despite those challenges, the tone of the meeting was strikingly forward-looking, with several take-home messages for sponsors.

Patient input belongs at the beginning of development. FDA emphasized that patient input can help identify the outcomes that matter, inform trial design and recruitment, support fit-for-purpose clinical outcome assessments, and ultimately inform the benefit-risk assessment. That point carries particular weight here. Patient voices helped bring FDA to the table in the first place. Continued patient engagement may also be essential to designing studies capable of delivering the evidence FDA needs.

Dose-ranging and exposure-response work also deserve substantial attention before pivotal trials. Although these are familiar components of drug development, testosterone presents added challenges: there is no well-established therapeutic testosterone concentration for women, the relationship between serum concentrations and clinical benefit remains uncertain, and the lowest effective dose may differ by indication. Sponsors will need to think just as carefully about trial design and endpoint performance, particularly in settings susceptible to substantial placebo response. Assay strategy matters, particularly given the challenges of reliably measuring the lower testosterone concentrations generally observed in women. And defining requirements and planning for long-term safety cannot be an afterthought.

Perhaps most importantly, FDA repeatedly invited sponsors to engage with the Agency. FDA officials encouraged developers to discuss protocols, endpoints, assays, dose selection, and approaches to addressing long-term safety throughout development. The Agency reiterated the same message on multiple occasions: come to them early and often.

A Call to Action

The workshop ended where it began: with the need for more evidence.

FDA officials described the meeting as a “call to action” and expressed hope that it would catalyze sponsors to undertake the research needed to move the field forward.

But the call to action should not be directed at industry alone. The scientific and regulatory challenges discussed throughout the day will require continued engagement from FDA as well. The Agency has an important role to play in helping translate the questions identified at the workshop into workable development paths, including providing clarity around acceptable endpoints, dose-finding strategies, safety expectations, and approaches to filling longstanding evidence gaps.

We are excited to see what FDA, industry, and the women who started this conversation do next.