Merck KgaA, Pfizer Receive FDA Orphan Drug Status For Avelumab In MCC

(RTTNews) - German drug maker Merck KGaA (MKGAY) and U.S. drug maker Pfizer Inc. (PFE) said Friday that the U.S. Food and Drug Administration has granted Orphan Drug Designation for their investigational cancer immunotherapy, avelumab, for the treatment of Merkel cell carcinoma or MCC, a rare and aggressive type of skin cancer.

Avelumab, also referred to as MSB0010718C, is an investigational fully human monoclonal IgG1 antibody against programmed death-ligand 1 or anti-PD-L1. Avelumab is under clinical investigation and has not been proven to be safe and effective.

Merck KGaA 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.

The FDA grants Orphan Drug Designation status to drugs that are intended to treat rare diseases or disorders that affect fewer than 200,000 people in the U.S., or those that affect more than 200,000 people, but are unlikely to recover the costs of developing and marketing the drug. The orphan drug designation for avelumab applies only to MCC.

Dr. Luciano Rossetti, Head of Global Research & Development at Merck's biopharmaceutical business, Merck Serono said, "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. 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."

JAVELIN Merkel 200 is a multicenter, single-arm, open-label study. The primary endpoint of the study is objective response rate. 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.

MCC is a rare and aggressive disease in which cancer cells form in the top layer of the skin, close to nerve endings. Each year, there are about 1,500 new cases of MCC diagnosed in the U.S.

MCC, also known as neuroendocrine carcinoma of the skin or trabecular cancer, often starts in those areas of skin that are most often exposed to the sun, including the head and neck, arms, legs, and trunk. Treatment for metastatic or Stage IV MCC is generally palliative.

PFE is trading at $32.58, down $0.12 or 0.37 on a volume of 4.16 million shares.

In Frankfurt, Merck shares are trading at 80.31 euros, up 1.77 euros or 2.25 percent on a volume of 381,014 shares.