Polycystic Ovary Syndrome (PCOS): From diagnosis to treatments and the future

Which symptoms can occur with PCOS?

PCOS presents with a range of symptoms that can vary considerably between individuals. The most frequently observed symptoms include:

• Irregular Menstrual Cycles: It is common for women with PCOS to experience menstrual cycles that are infrequent, irregular, or prolonged.
• Hyperandrogenism: Elevated levels of androgens* can result in the development of symptoms such as hirsutism (growth of excessive male-pattern hair on face and body), severe acne, and male-pattern baldness.
• Polycystic Ovaries: PCOS is characterized by the presence of multiple small cysts within the ovaries. These fluid-filled sacs do not regularly release eggs, meaning those remain trapped within the ovaries. The affected ovary may exhibit slight enlargement, and the condition may manifest in both ovaries simultaneously.
• Weight Fluctuations and Pre-diabetes: Many women with PCOS gain weight, especially around the abdomen, and may struggle with weight fluctuations due to hormonal imbalances. They are also at higher risk of developing pre-diabetes, where blood sugar levels are higher than normal but not yet diabetes. This is often caused by insulin resistance and can lead to type 2 diabetes if not managed early.
• Infertility: Irregular ovulation is a common cause of infertility in individuals with PCOS.

Other potential symptoms include the presence of dark patches of skin (acanthosis nigricans), fatigue, and mood disorders such as depression and anxiety.

How is PCOS currently diagnosed?

A diagnosis of PCOS is typically made using a combination of medical history, physical examination, and diagnostic testing. The most widely used criteria are the Rotterdam criteria, which require at least two of the following three features:

• Hyperandrogenism: This refers to high levels of male hormones, which can lead to symptoms like acne or excess facial and body hair, and can be confirmed through blood tests.
• Irregular or Absent Menstrual Periods: This includes infrequent or very long menstrual cycles.
• Polycystic Ovaries: An ultrasound may show multiple small cysts on the ovaries.

However, PCOS remains underdiagnosed in approximately 70% of cases¹World Health Organization : WHO & World Health Organization : WHO. (2025, 7 février). Polycystic ovary syndrome. https://www.who.int/news-room/fact-sheets/detail/polycystic-ovary-syndrome. Many women face long delays before receiving a diagnosis, often due to a lack of awareness among healthcare providers or the wide variation in how symptoms present. This diagnostic gap can lead to years of unmanaged symptoms and health risks.

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Current Treatment Options

Currently, there are no treatments available that target the underlying causes of PCOS. The current approach to PCOS management is primarily symptom-oriented, with the objective of reducing the risk of long-term complications. The specific treatment plan is determined on an individual basis, considering the symptoms and concerns of the patient. Treatment options include:

Lifestyle Modifications

A balanced diet and regular exercise can facilitate weight loss, which in turn can improve insulin sensitivity, reduce androgen levels and regulate menstrual cycles. Even modest weight loss (5-10% of body weight) can have significant benefits.

Medications

• Hormonal Contraceptives: Combined oral contraceptives containing both oestrogen and progestin can regulate menstrual cycles, reduce androgen levels and alleviate symptoms such as acne and excessive hair growth. For women who are unable to take oestrogen, progestin-only options can assist in regulating menstrual cycles and in the prevention of endometrial cancer. These hormonal contraceptives are the first-line therapies administered for PCOS.
• Anti-Androgens: Medications such as spironolactone have been demonstrated to reduce the growth of excess hair and the occurrence of acne by blocking the effects of androgens.
• Metformin: A commonly prescribed medication for the treatment of type 2 diabetes, metformin has been demonstrated to enhance insulin sensitivity and may facilitate weight loss and ovulation regulation.
• Fertility Medications: For women presenting with fertility issues, medications such as clomiphene citrate or letrozole have been shown to stimulate ovulation. In cases where these are ineffective, injectable gonadotropins (follicle stimulating hormones, FHS) or in vitro fertilisation (IVF) may be considered as potential treatment options.

Other Treatments

• Hair Removal: Techniques like laser hair removal or electrolysis can manage excessive hair growth.
• Acne Treatments: Topical or oral medications may be prescribed to address severe acne.

Future Trends and Drugs in Development

As the understanding of PCOS advances, so do the methodologies deployed for its management and treatment. The current research focus is on developing new or adapting existing treatment modalities and targeted therapies that address the underlying mechanisms of PCOS.

Emerging Therapies

1. GnRH antagonists: Drugs such as Elagolix (by Abbvie Inc.), are currently being investigated for the treatment of PCOS. These agents inhibit the release of FSH and lutenizing hormones (LH), but Phase II²Snabes, M. C., Ng, J., Li, H., Ali, I., Shebley, M., & Schlaff, W. D. (2023). Phase 2, double-blind, randomized, placebo-controlled study of the safety and efficacy of elagolix in women with polycystic ovary syndrome. F&S Reports, 4(2), 206‑212. https://doi.org/10.1016/j.xfre.2023.02.007 trials have yielded limited success in achieving the normalisation of ovulatory cycles. While Elagolix alone has been observed to have minimal efficacy in the induction of follicular development and menstrual cycle regulation, hopes are high for future research prioritising the exploration of alternative dosing regimens and combination therapies.
2. Neurokinin B receptor antagonism: Phase II³Fraser, G. L., Obermayer-Pietsch, B., Laven, J., Griesinger, G., Pintiaux, A., Timmerman, D., Fauser, B. C. J. M., Lademacher, C., Combalbert, J., Hoveyda, H. R., & Ramael, S. (2021). Randomized Controlled Trial of Neurokinin 3 Receptor Antagonist Fezolinetant for Treatment of Polycystic Ovary Syndrome. The Journal Of Clinical Endocrinology & Metabolism, 106(9), e3519‑e3532. https://doi.org/10.1210/clinem/dgab320 studies of the NK3 receptor antagonist Fezolinetant (Astellas Pharma) demonstrated its efficacy in reducing serum LH and testosterone levels, as well as the LH-to-FSH ratio in patients with PCOS. This suggests the potential for kisspeptin-neurokinin B (NKB) antagonism as a treatment option, although further research is required to confirm its efficacy and to elucidate its clinical implications.
3. Sodium-glucose co-transporter type 1 and 2 (SGLT1/2): The use of SGLT1/2 inhibitors such as licogliflozin (Novartis) as treatments for insulin resistance has demonstrated efficacy in the alleviation of PCOS symptoms through the reduction of glucose, insulin, and androgen levels. Phase II⁴Tan, S., Ignatenko, S., Wagner, F., Dokras, A., Seufert, J., Zwanziger, D., Dunschen, K., Zakaria, M., Huseinovic, N., Basson, C. T., Mahling, P., Fuhrer, D., & Hinder, M. (2021). Licogliflozin versus placebo in women with polycystic ovary syndrome : A randomized, double‐blind, phase 2 trial. Diabetes Obesity And Metabolism, 23(11), 2595‑2599. https://doi.org/10.1111/dom.14495 trials indicate that licogliflozin effectively lowers glucose, insulin, A4, and DHEAS levels in patients with PCOS, suggesting the potential for SGLT1/2 inhibitors as a treatment option. However, further research is required to confirm these findings and assess their impact on ovulation and overall clinical outcomes.
4. Artemisinin: Popularly known as an anti-Malaria drug, artemisinin has previously been investigated for its potential effects on adipose tissue in murine models. Additionally, researchers from the University of Shanghai have identified symptom-reducing effects in mice with a PCOS-comparable disease. This prompted the commencement of a preliminary study, the results of which indicated that artemisinin was effective in reducing androgen and AMH levels, as well as promoting the regularity of menstrual cycles in women with PCOS.

Future Direction – Improved Diagnostic Tools

The future of PCOS diagnosis is shifting toward more accessible and less invasive testing methods. Roche Diagnostics has developed the Elecsys AMH Plus immunoassay, a blood test that measures anti-Müllerian hormone (AMH) levels⁵Mihaila, S., & Mihaila, S. (2024, 12 février). Could Roche’s test change the way we diagnose PCOS ? – FemTech World. FemTech World. https://www.femtechworld.co.uk/news/can-roches-test-change-the-way-we-diagnose-pcos-heres-what-doctors-think/. Elevated AMH levels are indicative of a key marker of PCOS. This test offers a non-invasive alternative to traditional transvaginal ultrasounds, aligning with the updated 2023 Rotterdam Criteria and potentially improving early diagnosis rates.

Other companies are also advancing PCOS diagnostics. Metabolon, a U.S.-based firm, received a National Institutes of Health award in December 2023 to develop a metabolomics-based PCOS diagnostic test, aiming to identify unique metabolic biomarkers associated with the condition.⁶Deswal, P. (2023, 6 décembre). Metabolon scores NIH award to develop PCOS diagnostics. Medical Device Network. https://www.medicaldevice-network.com/news/metabolon-scores-nih-award-to-develop-pcos-diagnostics/

These innovations represent a significant step forward in addressing the underdiagnosis of PCOS, which affects up to 70% of women with the condition.⁷World Health Organization : WHO & World Health Organization : WHO. (2025b, février 7). Polycystic ovary syndrome. https://www.who.int/news-room/fact-sheets/detail/polycystic-ovary-syndrome By simplifying the diagnostic process and making it more patient-friendly, these advancements hold promise for earlier detection and improved management of PCOS.

Polycystic Ovary Syndrome (PCOS) is a complex endocrine disorder, characterised by a broad spectrum of symptoms and associated complications. Although current treatments are primarily focused on managing symptoms and improving quality of life, ongoing research and drug development may offer promising prospects for more effective, disease-modifying therapies and management strategies. Alcimed can help you with your projects in these areas. Don’t hesitate to contact our team!

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About the authors:

Mikka, Senior Consultants in Alcimed’s Healthcare Team in Germany.

Lisa, Senior Consultants in Alcimed’s Healthcare Team in Germany.

* Androgens are hormones that control the development of male sexual characteristics.