Avelumab Receives Orphan Drug Designation for the Treatment of Merkel Cell Carcinoma

Avelumab, an investigational fully human monoclonal IgG1 antibody against programmed death-ligand 1 (anti-PD-L1), is receiving orphan drug designation by the US Food and Drug Administration (FDA) for the treatment of Merkel cell carcinoma (MCC)-also called neuroendocrine carcinoma of the skin. This experimental agent has been shown to be effective in combating other tumor types, and now it may soon become a new treatment option for this rare and aggressive type of skin cancer.  

On September 25, 2015, Merck KGaA and Pfizer announced the new designation for avelumab calling it an important regulatory milestone.

“We continue to dedicate significant resources to accelerate our clinical trial program, with a goal of helping patients who are fighting rare and difficult-to-treat diseases, such as Merkel cell carcinoma,” said Luciano Rossetti, MD, Global Head of Research & Development of the biopharmaceutical business of Merck KGaA, Darmstadt, Germany, in a press release.

Currently, this agent is being assessed in a phase II study (JAVELIN Merkel 200). It is a multicenter, single-arm, open-label study evaluating 88 patients with metastatic MCC who have previously received at least one line of chemotherapy. The primary endpoint is objective response rate. The secondary endpoints include duration of response, progression-free survival (PFS), overall survival (OS), and safety. The study is being conducted in sites across Asia Pacific, Australia, Europe, and North America.  

The clinical development program for avelumab now includes more than 1,000 patients treated across more than 15 tumor types, including NSCLC, breast cancer, gastric cancer, ovarian cancer, urothelial cancer, esophageal cancer, squamous cell carcinoma of the head and neck, renal cell carcinoma, MCC, melanoma, and mesothelioma. It is currently estimated there are 1,500 new cases of MCC diagnosed each year in the US. The current treatment options for MCC include surgery, radiation, and chemotherapy; treatment for metastatic or stage IV MCC is generally palliative.  

Avelumab is thought to potentially enable the activation of T cells and the adaptive immune system by inhibiting PD-L1 interactions. “There is a strong scientific and clinical rationale that by harnessing the body’s immune system, we may be able to control the disease and provide hope to patients fighting Merkel cell carcinoma,” said Mace Rothenberg, MD, who is Senior Vice President of Clinical Development and Medical Affairs and Chief Medical Officer for Pfizer Oncology, New York.