FDA Actions & Updates

The latest approvals, designations, and new indications from the U.S. Food and Drug Administration for oncology drugs.

Thursday, April 6, 2017

FDA Approves First Treatment for Rare Form of Skin Cancer

Avelumab has been granted accelerated approval by the FDA for the treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC), including those who have not received prior chemotherapy.

This is the first FDA-approved treatment for metastatic MCC. Avelumab targets the PD-1/PD-L1 pathway; by blocking these interactions, avelumab may help the body's immune system attack cancer cells.

"While skin cancer is one of the most common cancers, patients with a rare form called Merkel cell cancer have not had an approved treatment option until now," said Richard Pazdur, MD, Acting Director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research and Director of the FDA's Oncology Center of Excellence. "The scientific community continues to make advances targeting the body's immune system mechanisms for the treatment of various types of cancer. These advancements are leading to new therapies—even in rare forms of cancer where treatment options are limited or non-existent."

According to the NCI, approximately 1,600 people in the U.S. are diagnosed with MCC every year. While the majority of patients present with localized tumors that can be treated with surgical resection, approximately half of all patients will experience recurrence, and more than 30 percent will eventually develop metastatic disease. In patients with metastatic MCC, the cancer has spread beyond the skin into other parts of the body. 

The approval of avelumab was based on data from a single-arm trial of 88 patients with metastatic MCC who had been previously treated with at least one prior chemotherapy regimen. The trial measured the percentage of patients who experienced complete or partial shrinkage of their tumors (overall response rate) and, for patients with a response, the length of time the tumor was controlled (duration of response). Of the 88 patients who received avelumab in the trial, 33 percent experienced complete or partial shrinkage of their tumors. The response lasted for more than six months in 86 percent of responding patients and more than 12 months in 45 percent of responding patients.

Common side effects of avelumab include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reactions, rash, decreased appetite, and peripheral edema. The most common serious risks of avelumab are immune-mediated, such as pneumonitis, hepatitis, colitis, endocrinopathies, and nephritis. In addition, there is a risk of serious infusion-related reactions. Patients who experience severe or life-threatening infusion-related reactions should stop using avelumab. Women who are pregnant or breastfeeding should not take avelumab because it may cause harm to a developing fetus or a newborn baby.

The FDA granted this application Priority Review and Breakthrough Therapy designation; Avelumab also received Orphan Drug designation.​