FDA Workshop Signals Potential Approval Pathway for Testosterone Therapy in Menopausal Women

The U.S. Food and Drug Administration (FDA) held a workshop on September 17, 2026, regarding testosterone use in menopausal women following an August 18, 2026, notice published in the Federal Register (91 FR 53417). This Holland & Knight alert summarizes key developments from the workshop, examines the current off-label prescribing landscape, reviews the scientific evidence and gaps discussed at the workshop, and outlines the FDA's expectations for a regulatory approval pathway. For providers who prescribe or are considering prescribing testosterone to menopausal patients, the regulatory, evidentiary and informed-consent issues to monitor are provided.

Current Prescribing Landscape: How Providers Are Using Testosterone Off-Label

There is no FDA-approved testosterone product indicated for use in women. The FDA's decision to host a public meeting and open a federal docket reflects the agency's interest in expanding indications for testosterone use in women.

Unlike in the U.S., testosterone is currently approved for hypoactive sexual desire disorder (HSDD) in postmenopausal women in Australia, New Zealand, the United Kingdom and South Africa. The 2019 Global Consensus Position Statement, endorsed by 11 major medical societies, established that the only evidence-based indication for testosterone therapy in women is HSDD in postmenopausal women. The consensus statement also noted that safety data beyond 24 months is limited, particularly regarding cardiovascular and breast cancer risks, and that recommendations apply specifically to physiologic, not supraphysiologic, doses.

Nevertheless, off-label prescribing of testosterone for women nearing or past menopause has tripled over the past five years (although, in absolute numbers, less than 0.36 percent of menopausal women are prescribed testosterone), driven largely by the growth of the wellness and hormone optimization movement. Practitioners increasingly prescribe off-label FDA-approved testosterone products approved for use only in men, as well as compounded creams, gels, injectables and pellets, to address symptoms such as low libido, fatigue, cognitive changes and musculoskeletal decline. Off-label prescribing alone creates a lack of standardized dosing, quality control and long-term safety data. Pellet implants can deliver testosterone at supraphysiologic levels, which cannot be reversed if adverse side effects occur. In some settings, patients receive these products without ongoing clinical supervision.

FDA Workshop: Key Themes and Takeaways

The FDA workshop was jointly hosted by the FDA's Office of Women's Health (OWH) and Center for Drug Evaluation and Research (CDER). Participants included endocrinologists, urologists, gynecologists, patient advocates, and clinical leaders and medical officers from OWH and CDER. The workshop was organized into three substantive sessions, each addressing critical dimensions of the testosterone-in-women landscape.

Session I: Testosterone Physiology and Measurement Challenges

Key presenters included a professor of medicine specializing in integrative physiology and endocrinology and a leading endocrinologist from a major academic medical center. Notable points included:

• Unlike estrogen, testosterone does not drop dramatically during menopause, complicating the clinical framework for deficiency-based treatment.
• Presenters cautioned against the off-label use of testosterone for sexual dysfunction other than HSDD or for other purposes such as cognitive, cardiovascular, metabolic or bone health, noting that though testosterone may prove useful for these indications, the supporting data does not yet exist.
• Current testosterone assays lack the sensitivity and standardization needed for accurate measurement at the lower concentrations found in women.

Session II: Clinical Guidelines, Practice and Perspectives

This session brought together clinicians and patients to discuss the current state of practice. Presenters included the director of a major academic comprehensive menopause program, a urologist and prominent advocate for expanded testosterone access in women, and a patient perspective panel. The session was moderated by a deputy director from CDER's Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine. Among key themes:

• Clinicians presented compelling patient narratives involving pain during intercourse, low libido and hot flashes – symptoms of menopause that do not fully resolve with estrogen therapy alone.
• Adding testosterone to estrogen and progesterone therapy demonstrated meaningful symptomatic improvement in many patients.
• Clinicians emphasized that most male testosterone products are prescribed off-label and that an FDA-approved female-specific product would confer critical legitimacy on existing clinical practice.
• Clinicians also raised particular alarm about high-dose prescribing, explaining that proper informed consent is essential at these doses because long-term safety has not been studied.
• Clinicians further explained that there is no effective mechanism for reporting adverse effects from compounded and off-label testosterone products, further underscoring the safety risks.

Clinician presenters identified several major obstacles facing practitioners who currently prescribe testosterone to menopausal women:

• Formulation Variability. Without an FDA-approved product, clinicians must navigate different absorption rates, variable application sites and the inability to add bioavailability enhancers. Dispensing is inconsistent, and patients face difficulty obtaining reliable, reproducible dosing. An FDA-approved product would help standardize formulations and provide evidence-based dosing guidance.
• Compounding, Lack of Standardization and High Costs. Presenters noted that problems are associated with compounding practices, not with testosterone itself, including unreliable dosing. Compounding is generally discouraged by medical societies, and many prescribers are not comfortable prescribing testosterone to women when it is not FDA approved. Compounded products are also typically not covered by insurance, imposing significant out-of-pocket costs on patients. Further, the lack of standardization makes it difficult to provide patients long-term risk-benefit guidance.
• Controlled Substance Designation Barriers. Presenters also explained that testosterone's classification as a Schedule III controlled substance creates significant prescribing barriers. Clinicians require a U.S. Drug Enforcement Administration license to prescribe it. For telehealth practitioners, the controlled substance designation means prescriptions cannot be sent to states where the prescriber is not licensed, limiting access for patients in underserved areas. Pharmacists may refuse to dispense more than a six-month supply. Multiple workshop participants called for removal of the controlled substance designation for testosterone when prescribed to women at physiologic doses.

Session III: The FDA's Position on Safety and Efficacy

The FDA presenters in Session III provided a candid assessment of the current evidence base and the agency's safety concerns. The FDA noted that when it last comprehensively reviewed testosterone data approximately 20 years ago, it observed androgenic adverse effects, including acne, alopecia and mood changes, as well as metabolic effects such as weight gain and body composition changes.

Key data gaps identified by the FDA include:

• Cardiovascular Disease Risk. Cardiovascular disease is the leading cause of death in women. The FDA noted that existing studies have significant methodological limitations, including short study durations and a lack of randomization. The FDA concluded that available data are inadequate to assess cardiovascular risks associated with long-term testosterone use in women.
• Breast Cancer Risk. Breast cancer is the most common cancer and the second most common cause of death in postmenopausal women. Epidemiological studies suggest that endogenous testosterone may increase breast cancer risk, though some data suggests a potential protective benefit. The FDA noted renewed interest in understanding testosterone use among breast cancer patients but emphasized that the evidence remains inconclusive.
• Long-Term Safety Beyond 24 Months. Clinical data is limited by short trial durations. The FDA emphasized the need for longer-duration studies to establish a comprehensive safety profile.

Regulatory Pathway Signals

Perhaps the most significant outcome of the workshop was the series of signals the FDA provided regarding its approach to a potential testosterone approval pathway for women:

• Willingness to Engage with Drug Sponsors. The FDA explicitly stated that it "stands ready to have discussions with drug sponsors" and expressed its desire to "bring these products to American women." This represents a clear invitation for industry engagement.
• Potential to Leverage International Data. When asked whether manufacturers could rely on studies supporting approvals in other countries (e.g., the U.K.), the FDA acknowledged that it recognizes approved products in other jurisdictions and indicated there "may be ways to leverage existing information" but emphasized that this would require "additional agency discussions." The FDA cannot unilaterally accept a proprietary product's data package from outside the U.S. to support a domestic approval. Testosterone use is not the only therapeutic area where the FDA is considering using international data and approvals to potentially streamline bringing products to the U.S. market.
• Emphasis on Patient-Focused Outcome Measurement. The FDA underscored the importance of determining what is "meaningful to the patient" and what patients would consider a meaningful improvement. Clinical outcome assessments will be central to any successful submission.
• Dosing Research Is a Critical Priority. The FDA called for high-quality research on the lowest effective dose for each indication, the optimal dose for the right patient population and best delivery method. Inconsistent dosing was identified as a major barrier to both clinical practice and regulatory review.
• Defined Patient Populations and Clear Endpoints. The FDA emphasized that a compelling regulatory package must identify a specific patient population, articulate clear clinical endpoints and provide comprehensive data on formulation characteristics and baseline patient demographics.
• Concerns About Supraphysiologic Dosing. The FDA and clinical presenters highlighted risks associated with high-dose testosterone use (e.g., levels of 500-plus ng/dL), including unknown long-term effects, increases in estradiol and insufficient informed consent practices at these dose levels.

The FDA's closing statement, "Menopause is inevitable, but women should not have to suffer," reflects the agency's commitment to support and approve products addressing the needs of menopausal women. The convergence of clinical evidence, patient advocacy, international approvals and FDA engagement suggests that providers may have access to an FDA-approved testosterone product for their menopausal patients in the coming years, which would represent a significant improvement in the standard of care.

Drug developers should engage with the FDA if they have testosterone products in development, particularly for menopausal women. Clinicians should monitor FDA communications for follow-up guidance documents, draft frameworks or additional Federal Register notices that may emerge from this workshop and affect prescribing practices.

For more information or questions, please contact the authors.

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