Cyproterone Acetate Off-Label Prescribing in the US
Cyproterone acetate (CPA) is not FDA-approved and is not available for prescription in the United States, either on-label or off-label. 1
Regulatory Status in the United States
- CPA has no FDA approval for any indication in the US, making it unavailable through standard prescribing channels 1
- The drug is only available in Europe and Canada, where it is approved for androgen-sensitive metastatic prostate cancer 2, 3
- Unlike other medications that can be prescribed off-label after FDA approval for at least one indication, CPA cannot be prescribed at all in the US because it has never received FDA approval 1
Important Distinction: Off-Label vs. Non-Approved
- Off-label prescribing refers to using an FDA-approved medication for an indication not specifically approved by the FDA 4
- CPA does not fall into this category—it is a non-approved medication in the US market 1
- The only potential route for US access would be through FDA investigational drug protocols, similar to how domperidone is accessed for gastroparesis 4
Clinical Context and Alternatives
For Prostate Cancer:
- US clinicians must use alternative antiandrogens such as bicalutamide, enzalutamide, or apalutamide 2
- CPA's role in preventing tumor flare with LHRH agonist initiation is handled by other antiandrogens in US practice 2
For Transgender Healthcare:
- US providers use spironolactone or GnRH agonists as antiandrogen alternatives 2
- The World Professional Association for Transgender Health recognizes CPA as an option, but this is not applicable to US practice given its unavailability 2