Pembrolizumab
Pembrolizumab is a type of targeted therapy drug called an immune checkpoint inhibitor (a type of immunotherapy). It is a monoclonal antibody that binds to the protein PD-1 on the surface of immune cells called T cells. It works by keeping cancer cells from suppressing the immune system. This allows the immune system to attack and kill the cancer cells.
FDA label information for this drug is available at DailyMed.
Use in Cancer
Pembrolizumab is approved to treat:
- Biliary tract cancer that has spread or cannot be removed by surgery. It is used with gemcitabine hydrochloride and cisplatin.
- Breast cancer that is triple negative. Pembrolizumab is used:
- With chemotherapy in patients whose cancer has the PD-L1 protein and has come back and cannot be removed by surgery or has spread.
- With chemotherapy before surgery and then alone after surgery in patients with early-stage cancer that has a high risk of coming back.
- Cervical cancer. Pembrolizumab is used:
- Alone in patients whose cancer has come back or has spread to other parts of the body, has the PD-L1 protein, and got worse during or after chemotherapy.
- With chemotherapy, with or without bevacizumab, in patients whose cancer has the PD-L1 protein and has come back or has spread to other parts of the body or did not respond to treatment.
- Classic Hodgkin lymphoma. Pembrolizumab is used:
- In adults whose cancer does not respond to treatment or has relapsed.
- In children whose cancer is refractory or has relapsed after at least two other types of treatment.
- Cutaneous squamous cell carcinoma (a type of skin cancer) that has spread or come back. Pembrolizumab is used in patients whose cancer cannot be cured with surgery or radiation therapy.
- Endometrial carcinoma that cannot be cured with surgery or radiation therapy and that got worse after other systemic therapies. Pembrolizumab is used:
- With lenvatinib in patients whose cancer is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).
- Alone in patients whose cancer is MSI-H or dMMR.
- Esophageal or gastroesophageal junction cancer that has spread and cannot be cured with surgery or a combination of chemotherapy and radiation therapy. Pembrolizumab is used:
- With platinum chemotherapy and a fluoropyrimidine.
- Alone in patients with squamous cell cancer that has the PD-L1 protein and has been treated with systemic therapy.
- Gastric (stomach) cancer or gastroesophageal junction adenocarcinoma that has spread or cannot be treated with surgery. Pembrolizumab is used as the first treatment with:
- Trastuzumab, a fluoropyrimidine, and platinum chemotherapy in patients whose cancer has the PD-L1 protein and is HER2 positive.¹
- Fluoropyrimidine and platinum chemotherapy in patients whose cancer is HER2 negative.
- Hepatocellular carcinoma (a type of liver cancer) in patients who have been treated with sorafenib.¹
- Melanoma. Pembrolizumab is used in:
- Patients whose cancer cannot be removed by surgery or has spread to other parts of the body.
- Adults and children aged 12 years and older with stage IIB, stage IIC, or stage III melanoma. It is used to prevent melanoma from coming back after it has been removed by surgery.
- Merkel cell carcinoma in adults and children. Pembrolizumab is used in patients whose cancer has come back or spread.
- Microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) cancer that is has spread to other parts of the body or cannot be removed by surgery. Pembrolizumab is used to treat:
- Solid tumors in adults and children that got worse after other treatment and cannot be treated with other therapies.
- Colorectal cancer.
MSI-H and dMMR cancers have certain genetic mutations and may not respond to some types of treatment.
- Non-small cell lung cancer. Pembrolizumab is used:
- With pemetrexed and platinum chemotherapy as the first treatment in patients with nonsquamous cancer that has spread to other parts of the body and does not have a mutation in the EGFR gene or ALK gene.
- With carboplatin and either paclitaxel or paclitaxel albumin-stabilized nanoparticle formulation as the first treatment for squamous cancer that has spread to other parts of the body.
- With platinum chemotherapy before surgery and then alone as adjuvant therapy, to help keep the cancer from coming back after surgery.
- Alone as the first treatment in patients whose cancer has the PD-L1 protein and does not have a mutation in the EGFR gene or ALK gene. Pembrolizumab is used in patients with stage III cancer that cannot be treated with surgery, chemotherapy, or radiation therapy. It is also used in patients whose cancer has spread to other parts of the body.
- Alone in patients whose cancer has spread to other parts of the body, has the PD-L1 protein, and has gotten worse during or after treatment with platinum chemotherapy. Patients whose cancer has EGFR or ALK gene mutations should receive pembrolizumab only if their disease got worse after treatment with an FDA-approved therapy for these mutations.
- Alone in adults with stage IB, stage IIA, stage IIB, or stage IIIA cancer, to help keep the cancer from coming back after platinum chemotherapy and surgery.
- Primary mediastinal large B-cell lymphoma. Pembrolizumab is used in adults and children whose cancer does not respond to treatment or has returned after at least two other therapies.
- Renal cell carcinoma (a type of kidney cancer). It is used:
- With axitinib or lenvatinib as the first treatment in patients whose cancer has spread or cannot be removed by surgery.
- As adjuvant therapy in patients whose cancer has a high risk of coming back after surgery to remove the kidney or surgery to remove the kidney and metastatic cancer.
- Solid tumors that are tumor mutational burden-high (TMB-H) and have spread to other parts of the body or cannot be removed by surgery. Pembrolizumab is used in adults and children whose cancer got worse after treatment and who are not able to receive other therapies.¹
- Squamous cell carcinoma of the head and neck that has come back or has spread to other parts of the body. Pembrolizumab is used:
- With platinum chemotherapy and fluorouracil as the first treatment in patients whose cancer cannot be removed by surgery.
- Alone as the first treatment in patients whose cancer cannot be removed by surgery and whose tumors have the PD-L1 protein.
- Alone in patients whose cancer got worse during or after treatment with platinum chemotherapy.
- Urothelial carcinoma (a type of bladder cancer). Pembrolizumab is used:
- Alone in patients whose cancer has spread and cannot be treated with platinum chemotherapy or has gotten worse during or after treatment with platinum chemotherapy.
- Alone in patients with carcinoma in situ that did not respond to treatment with bacillus Calmette-Guérin (BCG) and has not spread to the bladder muscle. Pembrolizumab is used in patients whose disease cannot be treated with surgery or who have decided not to have surgery.
- With enfortumab vedotin in adults whose cancer has spread and cannot be treated with cisplatin.¹
¹This use is approved under FDA’s Accelerated Approval Program. As a condition of approval, confirmatory trial(s) must show that pembrolizumab provides a clinical benefit in these patients.
Pembrolizumab is also being studied in the treatment of other types of cancer.
More About Pembrolizumab
Definition from the NCI Drug Dictionary - Detailed scientific definition and other names for this drug.
MedlinePlus Information on Pembrolizumab - A lay language summary of important information about this drug that may include the following:
- warnings about this drug,
- what this drug is used for and how it is used,
- what you should tell your doctor before using this drug,
- what you should know about this drug before using it,
- other drugs that may interact with this drug, and
- possible side effects.
Drugs are often studied to find out if they can help treat or prevent conditions other than the ones they are approved for. This patient information sheet applies only to approved uses of the drug. However, much of the information may also apply to unapproved uses that are being studied.
Research Results and Related Resources
Groundbreaking Trial Results Expand Treatment Options for Some People with Bladder Cancer Effects
FDA Amends Approval of Pembrolizumab to Treat Stomach and Esophageal Junction Cancer
A Better Biomarker for Cancer Immunotherapy?
Rare Melanoma Very Likely to Respond to Treatment with Pembrolizumab
Immunotherapy’s Role in Treating Endometrial Cancer Expected to Grow
Targeted Therapy to Treat Cancer
Clinical Trials Accepting Patients
Find Clinical Trials for Pembrolizumab - Check for trials from NCI's list of cancer clinical trials now accepting patients.