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Merck KGaA, Darmstadt, Germany, and Pfizer Announce FDA Orphan Drug Designation for Investigational Immunotherapy Avelumab in Merkel Cell Carcinoma


Merck KGaA, Darmstadt, Germany, and Pfizer today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for the investigational cancer immunotherapy avelumab* for the treatment of Merkel cell carcinoma (MCC), a rare and aggressive type of skin cancer.1,2 Each year, there are approximately 1,500 new cases of MCC diagnosed in the US.3

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Avelumab (also referred to as MSB0010718C) is an investigational fully human monoclonal IgG1 antibody against programmed death-ligand 1 (anti-PD-L1). Merck KGaA, Darmstadt, Germany, and Pfizer are currently conducting a Phase II study (JAVELIN Merkel 200) to assess the safety and efficacy of avelumab in patients with metastatic MCC who have progressed after at least one prior chemotherapy regimen.

“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 Dr. Luciano Rossetti, Global Head of Research & Development of the biopharmaceutical business of Merck KGaA, Darmstadt, Germany. “It is encouraging to be included in the FDA’s orphan drug program as we eagerly await the results of our Phase II trial of avelumab in this deadly skin cancer.”

“This is a rare and lethal type of skin cancer, especially when it has progressed despite prior chemotherapy,” said Dr. Mace Rothenberg, Senior Vice President of Clinical Development and Medical Affairs and Chief Medical Officer for Pfizer Oncology. “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.”

JAVELIN Merkel 200 is a multicenter, single-arm, open-label study evaluating patients with metastatic MCC who have previously received at least one line of chemotherapy. The primary endpoint is objective response rate, and secondary endpoints include duration of response, progression-free survival, overall survival and safety. The study, which exceeded its expected enrollment of 84 patients with 88 patients enrolled, is being conducted in sites across Asia Pacific, Australia, Europe and North America.

The JAVELIN clinical trial program also includes a Phase III study in Stage IIIb/IV or recurrent non-small cell lung cancer (NSCLC) designed to assess the efficacy and safety of avelumab compared with docetaxel in patients who have experienced disease progression after receiving a prior platinum-containing doublet therapy (JAVELIN Lung 200); a Phase Ib, open-label, multicenter, multiple-dose trial designed to estimate the maximum tolerated dose and select the recommended Phase II dose of avelumab in combination with axitinib** in patients with previously untreated advanced renal cell carcinoma (RCC; JAVELIN Renal 100); an international Phase I open-label, multiple ascending dose trial to investigate the safety, tolerability, pharmacokinetics, biological and clinical activity in patients with metastatic or locally advanced solid tumors (JAVELIN Solid Tumor); and a Phase I trial to investigate the tolerability, safety, pharmacokinetics, biological, and clinical activity of avelumab in Japanese patients with metastatic or locally advanced solid tumors (JAVELIN Solid Tumor Japan) with an expansion cohort in Asian patients with gastric cancer.

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 (e.g., bladder), esophageal cancer, SCCHN, RCC, MCC, melanoma and mesothelioma.

About the FDA Orphan Drug Designation

FDA orphan drug designation is granted to drugs intended to treat rare diseases or disorders that affect fewer than 200,000 people in the US, or those that affect more than 200,000 people, but are unlikely to recover the costs of developing and marketing the drug. Orphan drug designation by the FDA qualifies...