The U.S. Food and Drug Administration (FDA) has approved the first immunotherapy regimen shown to extend survival in a subset of ovarian cancer patients.
The FDA authorized Keytruda (pembrolizumab), made by Merck, in combination with chemotherapy, for PD-L1–positive, platinum-resistant epithelial ovarian, fallopian tube, and primary peritoneal cancers. Keytruda plus weekly Taxol (paclitaxel), with or without Avastin (bevacizumab), is authorized for patients who have received one or two prior systemic treatments.
The decision is based on findings from the Phase III KEYNOTE-B96 study and marks the first time an immune checkpoint inhibitor regimen has demonstrated a significant overall survival benefit in a Phase III ovarian cancer trial. Previous immunotherapy studies in ovarian cancer have not shown this level of benefit. Although improvement was observed across the study population, the benefit was strongest in patients with tumors that tested PD-L1 positive, which is why the FDA approval is limited to this group.
How this immunotherapy works
Some tumors make a protein called PD-L1 that helps them hide from the immune system. PD-1 checkpoint inhibitor treatments work by blocking PD-L1, helping the immune system better recognize and attack cancer cells.
The FDA also approved a companion diagnostic test to help identify people with ovarian, fallopian tube, or primary peritoneal cancers whose tumors are PD-L1 positive. This test can help doctors decide whether this treatment may be a good option.
What This Means for the Ovarian Cancer Community
For many years, immunotherapy trials in ovarian cancer did not improve survival. This study represents an important milestone by identifying a group of patients—those with PD-L1–positive, platinum-resistant disease—who may benefit from this approach, offering a new treatment option for patients with historically limited choices.
For many patients with ovarian cancer, the disease can become platinum-resistant, at which point recurrence is not just a setback—it’s when options can become limited, and the reality patients face can change very quickly,” said Bradley Monk, MD, FACOG, FACS, gynecologic oncologist and Medical Director of the Late-Stage Clinical Research Program at Florida Cancer Specialists & Research Institute, in a press release.
For patients who have been previously treated with standard platinum-based therapies, the FDA approvals of these pembrolizumab-based regimens offer the possibility of more time.
What to Know
What was approved for ovarian cancer?
The FDA approved Keytruda (pembrolizumab) or Keytruda Qlex (pembrolizumab and berahyaluronidase alfa-pmph) in combination with weekly Taxol (paclitaxel), with or without Avastin (bevacizumab), for adults with PD-L1–positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received prior treatment.
Who is eligible for this immunotherapy?
The approval is limited to platinum-resistant recurrent ovarian, fallopian tube, or primary peritoneal cancer patients whose tumors test positive for PD-L1 using an FDA-authorized test. In the clinical trial, patients with PD-L1–positive tumors experienced the greatest benefit from the immunotherapy regimen.
What does platinum-resistant ovarian cancer mean?
Platinum-resistant ovarian cancer refers to disease that returns or progresses within six months after treatment with a platinum-based chemotherapy regimen. At this stage, treatment options are often more limited.
Is this the first immunotherapy approved for ovarian cancer?
Yes. This is the first immune checkpoint inhibitor regimen to show a significant overall survival benefit in a Phase III ovarian cancer trial, which led to FDA approval.
Should patients ask about PD-L1 testing?
PD-L1 testing can determine whether this treatment is an option. Patients should discuss testing and treatment options with their healthcare provider.
Note: This article is intended to share research and regulatory news. It is not medical advice. Patients should speak with their healthcare team about individual treatment decisions.
