First drug for rare skin cancer wins approval

FDA approved the first treatment for metastatic Merkel cell carcinoma (MCC), a rare, aggressive skin cancer.

The agency granted accelerated approval to avelumab (Bavencio, EMD Serono) for the treatment of adults and pediatric patients aged 12 years and older with MCC, including those who have not received prior chemotherapy.

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“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 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.”

Bavencio received accelerated approval, which enables FDA to approve drugs for serious conditions to fill an unmet medical need using clinical trial data that is thought to predict a clinical benefit to patients. Further clinical trials are required to confirm Bavencio’s clinical benefit, which are currently being conducted.

Around1,600 people in the US are diagnosed with MCC every year, according to the National Cancer Institute.

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Bavencio works by targeting the PD-1/PD-L1 pathway (proteins found on the body’s immune cells and some cancer cells). By blocking these interactions, Bavencio may help the body’s immune system attack cancer cells.

The drug’s approval was based on data from a single-arm trial of 88 patients with metastatic MCC who had been previously treated with at least 1 prior chemotherapy regimen. Of the 88 patients who received Bavencio in the trial, 33% experienced complete or partial shrinkage of their tumors. The response lasted for more than six months in 86% of responding patients, and more than 12 months in 45% of responding patients.

Common side effects of Bavencio include fatigue, musculoskeletal pain, diarrhea, nausea, infusion-related reactions, rash, decreased appetite and swelling of the limbs (peripheral edema).

The most common serious risks of Bavencio are immune-mediated, where the body’s immune system attacks healthy cells or organs, such as the lungs (pneumonitis), liver (hepatitis), colon (colitis), hormone-producing glands (endocrinopathies) and kidneys (nephritis). In addition, there is a risk of serious infusion-related reactions, and patients who experience severe or life-threatening infusion-related reactions should stop using Bavencio. Women who are pregnant or breastfeeding should not take Bavencio because it may cause harm to a developing fetus or a newborn baby.

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