FDA Panel Recommends Limiting Immunotherapy Access for Stomach and Esophageal Cancer Patients Based on PD-L1 Expression

FDA Advisory Panel Vote:
The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted to limit the use of immunotherapies Keytruda (pembrolizumab) and Opdivo (nivolumab) for patients with HER2-negative advanced gastroesophageal junction cancer and esophageal squamous cell carcinoma, recommending they be available only to patients whose cancer expresses PD-L1 at levels equal to or greater than 1%.

Background:
The FDA originally approved these immunotherapies for all patients regardless of PD-L1 expression in 2021. However, subsequent studies showed that patients with higher PD-L1 expression had better outcomes, leading to concerns about the risk-benefit profile for patients with low or no PD-L1 expression.

Patient and Advocate Testimony:
Patients and advocates, including ECAN’s CEO Mindy Mintz Mordecai and esophageal cancer patient Kimberly Wilson, testified against restricting access, citing concerns about the reliability of PD-L1 scoring and the potential loss of hope for some patients.

Compassionate Use:
Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence, suggested that drugmakers extend compassionate use or expanded use protocols to provide the immunotherapies free of charge to PD-L1 negative patients who desire the treatment.

Future Decisions:
The FDA is expected to decide in December whether to approve Tevimbra (tislelizumab) for use in these patients, with the recommendation to restrict its use based on PD-L1 expression levels also applying to this drug.