On November 30, 2015 Merck KGaA, Darmstadt, Germany, and Pfizer reported that the European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion for Orphan Drug designation (ODD) for avelumab*, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, pending an official decision by the European Commission (EC), expected in December (Press release, Pfizer, NOV 30, 2015, View Source [SID:1234508363]). Schedule your 30 min Free 1stOncology Demo! The COMP positive opinion is for the 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 2,500 new cases of MCC diagnosed in the European Union (EU).3 There is currently no therapy approved specifically for the treatment of metastatic MCC.4
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"While significant therapeutic advances have been made against other types of skin cancer, similar progress has not been made against Merkel cell carcinoma. There is a great need for effective therapies in this disease," said Dr. Mace Rothenberg, Senior Vice President of Clinical Development and Medical Affairs and Chief Medical Officer for Pfizer Oncology. "Orphan Drug Designation is an important regulatory tool that can help facilitate development of a new treatment option for patients in Europe with this serious and rare condition."
The COMP’s positive opinion follows the US Food and Drug Administration’s ODD for avelumab for the treatment of MCC that was received in September, Fast Track designation for avelumab for the treatment of metastatic MCC that was received in October, and Breakthrough Therapy Designation for avelumab for the treatment of metastatic MCC that was received in November. In order for a drug to be granted ODD by the EMA, it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development; and where no satisfactory treatment is currently available.
"We are delighted the EMA’s Committee for Orphan Medicinal Products has considered that avelumab matches the Orphan Drug designation criteria for metastatic Merkel cell carcinoma in the EU," said Dr. Luciano Rossetti, Head of Global Research & Development at Merck KGaA, Darmstadt, Germany’s biopharma business. "We look forward to working closely with the EMA to make this potential treatment available to patients as soon as possible, and we eagerly await the results of our Phase II trial in this rare skin cancer."
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 whose disease has progressed after at least one prior chemotherapy regimen. JAVELIN Merkel 200 is a multicenter, single-arm, open-label Phase II study with a primary objective of objective response rate.
The clinical development program for avelumab now includes more than 1,400 patients who have been treated across more than 15 tumor types, including breast cancer, gastric/gastro-esophageal junction cancers, head and neck cancer, MCC, mesothelioma, melanoma, non-small cell lung cancer, ovarian cancer, renal cell carcinoma and urothelial (e.g. bladder) cancer.
About the EMA Orphan Drug Designation
An ODD by the EMA allows a pharmaceutical company to benefit from incentives from the EU to develop a medicine for a rare disease. Applications for ODD are examined by the COMP, which adopts an opinion that is forwarded to the EC. The EC then decides whether to grant an orphan designation for the medicine in question within 30 days of receipt of the COMP opinion.
Pharmaceutical companies that obtain ODD benefit from a number of incentives, including protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity once the medicine is on the market. Fee reductions are also available, depending on the status of the sponsor and the type of service required.
*Avelumab is the proposed International Nonproprietary Name for the anti-PD-L1 monoclonal antibody (MSB0010718C). Avelumab is under clinical investigation and has not been proven to be safe and effective. There is no guarantee any product will be approved in the sought-after indication by any health authority worldwide.
References
1. Hughes MP et al. Merkel cell carcinoma: epidemiology, target, and therapy. Curr Dermatol Rep 2014;3:46–53.
2. Kaae J et al. Merkel cell carcinoma: incidence, mortality, and risk of other cancers. J Natl Cancer Inst 2010;102(11):793–801.
3. IMMOMEC (European Commission). Merkel cell carcinoma. Available at: View Source (link is external) Last accessed November 2015
4. Miller NJ et al. Emerging and mechanism-based therapies for recurrent or metastatic Merkel cell carcinoma. Curr Treat Options Oncol; 2013 14(2): 249–6
About Merkel Cell Carcinoma (MCC)
MCC is a rare and aggressive disease in which cancer cells form in the top layer of the skin, close to nerve endings. MCC, which is 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. Risk factors for MCC include sun exposure and having a weak immune system (i.e., solid organ transplant recipients, people with HIV/AIDS and people with other cancers, such as chronic lymphocytic leukemia, are at higher risk). Caucasian males over age 50 are at increased risk. MCC tends to metastasize at an early stage, spreading initially to nearby lymph nodes, and then potentially to more distant areas in the body, including other lymph nodes or areas of skin, lungs, brain, bones or other organs. Current treatment options for MCC include surgery, radiation and chemotherapy. Treatment for metastatic or Stage IV MCC is generally palliative.
About Avelumab
Avelumab (also known as MSB0010718C) is an investigational fully human anti-PD-L1 IgG1 monoclonal antibody. By inhibiting PD-L1 interactions, avelumab is thought to potentially enable the activation of T-cells and the adaptive immune system. By retaining a native Fc-region, avelumab is thought to engage the innate immune system and induce antibody-dependent cell-mediated cytotoxicity (ADCC). In November 2014, Merck KGaA, Darmstadt, Germany, and Pfizer announced a strategic alliance to co-develop and co-commercialize avelumab.
Alliance between Merck KGaA, Darmstadt, Germany, and Pfizer Inc., New York, US