Short answer: No drug is FDA approved specifically for PCOS, so the medicines used for it are prescribed off-label. The 2023 international guideline says metformin should be considered for metabolic goals in adults with a BMI of 25 or more. It says GLP-1 drugs could be considered for higher weight, as for anyone else. Inositol "could be considered", but a 30-trial review called its evidence limited and inconclusive.
| Compound | Best evidence | What it shows |
|---|---|---|
| Metformin | Approved for a different use | Approved for type 2 diabetes. The 2023 PCOS guideline recommends considering it for weight and metabolic outcomes in adults with a BMI of 25 or more, with low-certainty evidence. |
| Liraglutide 3 mg | Human trials | In a 32-week trial of 82 women with obesity and PCOS, weight fell 5.7% vs 1.4% on placebo and free androgen index dropped. Stomach side effects hit 58% vs 19%. |
| Semaglutide (Wegovy) | Approved for a different use | Approved for weight and heart risk, not PCOS. The guideline lists it among anti-obesity drugs that could be considered for higher weight in adults with PCOS. |
| Inositol (myo- or D-chiro-) | Human trials | A review of 30 trials (2,230 people) found possible benefit on some metabolic measures and little else. The authors called the evidence limited and inconclusive. |
"Animal or lab studies only" means no published human trial for this goal that we could find. Results in rats or cells often do not carry over to people. Compounds that are not FDA-approved have no tested label for dose, purity or safety.
MedlinePlus describes PCOS as a hormonal disorder that affects the ovaries and many other parts of the body. The 2023 guideline diagnoses it in adults with two of three features: irregular cycles or ovulation problems, high androgens (male-type hormones) on a blood test or as signs like excess hair, and either a high AMH (anti-Müllerian hormone) level or many follicles on ultrasound.
In a recent international consensus endorsed by 56 organizations, the condition was renamed polyendocrine metabolic ovarian syndrome (PMOS). A 2026 report in AJOG explains that the "cysts" are arrested follicles, not true cysts, and that the condition is a lifelong hormone and metabolic problem, not only a fertility one. The diagnostic criteria did not change. It affects about 10% to 13% of women of reproductive age.
The guideline says it directly: medical therapy is generally not approved for use specifically in PCOS, so recommended use is evidence based but off-label. It asks clinicians to discuss the evidence, concerns and side effects with patients. The metformin label, for example, lists only type 2 diabetes.
The best placebo-controlled GLP-1 trial we found in PCOS tested liraglutide 3 mg for 32 weeks in 82 women with obesity, alongside a lifestyle program. Weight fell 5.7% on liraglutide vs 1.4% on placebo, and 25 of 44 vs 5 of 23 lost at least 5% of their weight. Free androgen index (a measure of active testosterone) fell on liraglutide. Stomach side effects were common: 58.2% vs 18.5%.
For inositol, the review written to inform the guideline pooled 30 trials. It found possible benefits for some metabolic measures, possible ovulation benefit from D-chiro-inositol, and likely no effect on other outcomes. Metformin may improve waist-to-hip ratio and excess hair more than inositol. Inositol likely causes fewer stomach side effects than metformin.
For trying to conceive, the same guideline makes letrozole the first-choice ovulation drug in PCOS, also off-label. It also notes that letrozole, metformin and clomiphene are off-label for ovulation induction in many countries.
Terms on this page: GLP-1 receptor agonist · Titration (dose escalation) · Half-life