FDA Approves Keytruda (pembrolizumab) for Advanced Non-Small Cell Lung Cancer
October 2, 2015 -- The U.S. Food and Drug Administration today granted accelerated approval for Keytruda (pembrolizumab) to treat patients with advanced (metastatic) non-small cell lung cancer (NSCLC) whose disease has progressed after other treatments and with tumors that express a protein called PD-L1. Keytruda is approved for use with a companion diagnostic, the PD-L1 IHC 22C3 pharmDx test, the first test designed to detect PD-L1 expression in non-small cell lung tumors.
Lung cancer is the leading cause of cancer death in the United States, with an estimated 221,200 new diagnoses and 158,040 deaths in 2015, according to the National Cancer Institute. NSCLC is the most common type of lung cancer.“Our growing understanding of underlying molecular pathways and how our immune system interacts with cancer is leading to important advances in medicine,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Today’s approval of Keytruda gives physicians the ability to target specific patients who may be most likely to benefit from this drug.”
Keytruda works by targeting the cellular pathway known as PD-1/PD-L1 (proteins found on the body’s immune cells and some cancer cells). By blocking this pathway, Keytruda may help the body’s immune system fight the cancer cells. In 2014, Keytruda was approved to treat patients with advanced melanoma following treatment with ipilimumab, a type of immunotherapy. Another drug, Opdivo (nivolumab), manufactured by Bristol-Meyers Squibb, also targets the PD-1/PD-L1 pathway and was approved to treat squamous non-small cell lung cancer (a certain kind of NSCLC) in 2015.
The safety of Keytruda was studied in 550 patients with advanced NSCLC. The most common side effects of Keytruda included fatigue, decreased appetite, shortness of breath or impaired breathing (dyspnea) and cough. Keytruda also has the potential to cause severe side effects that result from the immune system effect of Keytruda (known as “immune-mediated side effects”).
The effectiveness of Keytruda for this use was demonstrated in a subgroup of 61 patients enrolled within a larger multicenter, open-label, multi-part study. The subgroup consisted of patients with advanced NSCLC that progressed following platinum-based chemotherapy or, if appropriate, targeted therapy for certain genetic mutations (ALK or EGFR). This subgroup also had PD-L1 positive tumors based on the results of the 22C3 pharmDx diagnostic test. Study participants received 10 mg/kg of Keytruda every two or three weeks. The major outcome measure was overall response rate (percentage of patients who experienced complete and partial shrinkage of their tumors). Tumors shrank in 41 percent of patients treated with Keytruda and the effect lasted between 2.1 and 9.1 months.
In the 550 study participants with advanced NSCLC, severe immune-mediated side effects occurred involving the lungs, colon and hormone-producing glands. Other uncommon immune-mediated side effects were rash and inflammation of blood vessels (vasculitis). Women who are pregnant or breastfeeding should not take Keytruda because it may cause harm to a developing fetus or newborn baby. Across clinical studies, a disorder in which the body's immune system attacks part of the peripheral nervous system (Guillain-Barre Syndrome) also occurred.
The FDA granted Keytruda breakthrough therapy designation for this indication because Merck demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies. The drug also received priority review status, which is granted to drugs that, at the time the application was submitted, have the potential to be a significant improvement in safety or effectiveness in the treatment of a serious condition.
Keytruda was approved under the agency’s accelerated approval program, which allows the approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts confirmatory clinical trials. An improvement in survival or disease-related symptoms in patients being treated with Keytruda has not yet been established.
Keytruda is marketed by Merck & Co., based in Whitehouse Station, New Jersey and the PD-L1 IHC 22C3 pharmDx diagnostic test is marketed by Dako North America Inc. in Carpinteria, California.
Source: FDA
Posted: October 2015
Related articles
- FDA Approves Pembrolizumab for HER2 Positive Gastric or Gastroesophageal Junction Adenocarcinoma Expressing PD-L1 (CPS ≥1) - March 19, 2025
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Pemetrexed and Platinum Chemotherapy as First-Line Treatment for Adult Patients With Unresectable Advanced or Metastatic Malignant Pleural Mesothelioma (MPM) - September 18, 2024
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Carboplatin and Paclitaxel as Treatment for Adult Patients With Primary Advanced or Recurrent Endometrial Carcinoma - June 17, 2024
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Chemoradiotherapy as Treatment for Patients With FIGO 2014 Stage III-IVA Cervical Cancer - January 12, 2024
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Chemotherapy as First-Line Treatment for Locally Advanced Unresectable or Metastatic HER2-Negative Gastric or Gastroesophageal Junction (GEJ) Adenocarcinoma - November 16, 2023
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Gemcitabine and Cisplatin as Treatment for Patients With Locally Advanced Unresectable or Metastatic Biliary Tract Cancer - November 1, 2023
- FDA Approves Keytruda (pembrolizumab) for Treatment of Patients With Resectable NSCLC in Combination With Chemotherapy as Neoadjuvant Treatment, Then Single Agent as Adjuvant Treatment After Surgery - October 16, 2023
- FDA Approves Merck’s Keytruda (pembrolizumab) in Combination With Padcev (enfortumab vedotin-ejfv) for First-Line Treatment of Certain Patients With Locally Advanced or Metastatic Urothelial Cancer - April 3, 2023
- FDA Converts to Full Approval Indication for Keytruda (pembrolizumab) for Certain Adult and Pediatric Patients With Advanced Microsatellite Instability-High (MSI-H) or Mismatch Repair Deficient (dMMR) Solid Tumors - March 29, 2023
- FDA Approves Keytruda (pembrolizumab) as Adjuvant Treatment Following Surgical Resection and Platinum-Based Chemotherapy for Patients With Stage IB (T2a ≥4 Centimeters), II, or IIIA Non-Small Cell Lung Cancer (NSCLC) - January 27, 2023
- FDA Approves Merck’s Keytruda (pembrolizumab) for Patients With MSI‑H/dMMR Advanced Endometrial Carcinoma, Who Have Disease Progression Following Prior Systemic Therapy in Any Setting and Are Not Candidates for Curative Surgery or Radiation - March 21, 2022
- FDA Approves Merck’s Keytruda (pembrolizumab) as Adjuvant Therapy for Certain Patients With Renal Cell Carcinoma (RCC) Following Surgery - November 18, 2021
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Chemotherapy, With or Without Bevacizumab, as Treatment for Patients With Persistent, Recurrent or Metastatic Cervical Cancer Whose Tumors Express PD-L1 (CPS ≥1) - October 13, 2021
- FDA Approves Keytruda (pembrolizumab) Plus Lenvima (lenvatinib) Combination for First-Line Treatment of Adult Patients With Advanced Renal Cell Carcinoma (RCC) - August 11, 2021
- FDA Approves Keytruda (pembrolizumab) for Treatment of Patients With High-Risk Early-Stage Triple-Negative Breast Cancer in Combination With Chemotherapy as Neoadjuvant Treatment, Then Continued as Single Agent as Adjuvant Treatment After Surgery - July 27, 2021
- FDA Approves Keytruda (pembrolizumab) Plus Lenvima (lenvatinib) Combination for Patients With Certain Types of Advanced Endometrial Carcinoma - July 22, 2021
- FDA Approves Expanded Indication for Merck’s Keytruda (pembrolizumab) in Locally Advanced Cutaneous Squamous Cell Carcinoma (cSCC) - July 6, 2021
- FDA Approves Merck’s Keytruda (pembrolizumab) Combined With Trastuzumab and Chemotherapy as First-line Treatment in Locally Advanced Unresectable or Metastatic HER2-Positive Gastric or Gastroesophageal Junction Adenocarcinoma - May 5, 2021
- FDA Approves Merck’s Keytruda (pembrolizumab) Plus Platinum- and Fluoropyrimidine-Based Chemotherapy for Treatment of Certain Patients With Locally Advanced or Metastatic Esophageal or Gastroesophageal Junction (GEJ) Carcinoma - March 23, 2021
- FDA Approves Merck’s Keytruda (pembrolizumab) in Combination With Chemotherapy for Patients With Locally Recurrent Unresectable or Metastatic Triple‑Negative Breast Cancer Whose Tumors Express PD-L1 (CPS ≥10) - November 13, 2020
- FDA Approves Expanded Indication for Merck’s Keytruda (pembrolizumab) in Adult Patients With Relapsed or Refractory Classical Hodgkin Lymphoma (cHL) - October 15, 2020
- FDA Approves Merck’s Keytruda (pembrolizumab) for First-Line Treatment of Patients With Unresectable or Metastatic MSI-H or dMMR Colorectal Cancer - June 29, 2020
- FDA Approves Merck’s Keytruda (pembrolizumab) for the Treatment of Patients with Recurrent or Metastatic Cutaneous Squamous Cell Carcinoma (cSCC) that is Not Curable by Surgery or Radiation - June 24, 2020
- FDA Approves Second Biomarker-Based Indication for Merck’s Keytruda (pembrolizumab), Regardless of Tumor Type - June 17, 2020
- FDA Approves Merck’s Keytruda (pembrolizumab) for Use at an Additional Recommended Dose of 400 mg Every Six Weeks for All Approved Adult Indications - April 28, 2020
- FDA Approves Keytruda (pembrolizumab) for Patients With BCG-Unresponsive, High-Risk, Non-Muscle Invasive Bladder Cancer - January 8, 2020
- FDA Approves Keytruda (pembrolizumab) plus Lenvima (lenvatinib) Combination Treatment for Patients with Certain Types of Endometrial Carcinoma - September 17, 2019
- FDA Approves Keytruda (pembrolizumab) for Recurrent Locally Advanced or Metastatic Squamous Cell Carcinoma of the Esophagus - July 31, 2019
- FDA Approves Keytruda (pembrolizumab) for the Treatment of Metastatic Small Cell Lung Cancer (SCLC) - June 18, 2019
- FDA Approves Keytruda (pembrolizumab) for First-Line Treatment of Head and Neck Squamous Cell Carcinoma - June 11, 2019
- FDA Approves Keytruda (pembrolizumab) in Combination With Inlyta (axitinib) as First-Line Treatment for Patients With Advanced Renal Cell Carcinoma (RCC) - April 22, 2019
- FDA Approves Expanded Monotherapy Label for Merck’s Keytruda (pembrolizumab) for First-Line Treatment of NSCLC - April 11, 2019
- FDA Approves Keytruda (pembrolizumab) for the Adjuvant Treatment of Patients with Melanoma with Involvement of Lymph Node(s) Following Complete Resection - February 19, 2019
- FDA Approves Keytruda (pembrolizumab) for the Treatment of Patients with Recurrent Locally Advanced or Metastatic Merkel Cell Carcinoma - December 19, 2018
- FDA Approves Keytruda (pembrolizumab) for the Treatment of Patients with Hepatocellular Carcinoma (HCC) Who Have Been Previously Treated with Sorafenib - November 9, 2018
- FDA Approves Keytruda (pembrolizumab) in Combination with Carboplatin and Either Paclitaxel or Nab-Paclitaxel for the First-Line Treatment of Patients with Metastatic Squamous Non-Small Cell Lung Cancer (NSCLC) - October 30, 2018
- FDA Approves Expanded Label for Merck’s Keytruda (pembrolizumab) in Patients with Metastatic Nonsquamous NSCLC with No EGFR or ALK Genomic Tumor Aberrations - August 21, 2018
- FDA Approves Keytruda (pembrolizumab) for Treatment of Refractory or Relapsed Primary Mediastinal Large B-Cell Lymphoma (PMBCL) - June 13, 2018
- FDA Approves Keytruda (pembrolizumab) for Previously Treated Patients with Recurrent or Metastatic Cervical Cancer Whose Tumors Express PD-L1 - June 12, 2018
- FDA Approves Merck’s Keytruda (pembrolizumab) for Previously Treated Patients with Recurrent Locally Advanced or Metastatic Gastric or Gastroesophageal Junction Cancer Whose Tumors Express PD-L1 - September 22, 2017
- FDA Approves Keytruda (pembrolizumab) as First Cancer Treatment for any Solid Tumor with a Specific Genetic Feature - May 23, 2017
- FDA Approves Merck’s Keytruda (pembrolizumab) for Certain Patients with Locally Advanced or Metastatic Urothelial Carcinoma - May 18, 2017
- FDA Approves Merck’s Keytruda (pembrolizumab) as First-Line Combination Therapy for Patients with Metastatic Nonsquamous Non-Small Cell Lung Cancer (NSCLC), Irrespective of PD-L1 Expression - May 10, 2017
- FDA Approves Merck’s Keytruda (pembrolizumab) for Classical Hodgkin Lymphoma (cHL) - March 15, 2017
- FDA Approves Merck’s Keytruda (pembrolizumab) for First-Line Treatment of Certain Patients with Metastatic Non-Small Cell Lung Cancer - October 24, 2016
- FDA Approves Merck’s Keytruda (pembrolizumab) for Patients with Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma - August 5, 2016
- FDA Approves Expanded Indication for Keytruda (pembrolizumab) for the Treatment of Patients with Advanced Melanoma - December 18, 2015
- FDA Approves Keytruda (pembrolizumab) for Advanced Melanoma - September 4, 2014
- Merck to Present New Data in Five Tumor Types from Studies Evaluating Pembrolizumab - September 2, 2014
- Merck’s Investigational Anti-PD-1 Antibody, Pembrolizumab, Under Regulatory Review in Europe for Advanced Melanoma - June 30, 2014
Keytruda (pembrolizumab) FDA Approval History
More news resources
- FDA Medwatch Drug Alerts
- Daily MedNews
- News for Health Professionals
- New Drug Approvals
- New Drug Applications
- Drug Shortages
- Clinical Trial Results
- Generic Drug Approvals
Subscribe to our newsletter
Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.