EMA Advisory Committee Recommends Orphan Drug Status for Immunotherapy by Pfizer, Merck KGaA
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 being developed by Pfizer and Merck KGaA, pending an official decision by the European Commission, which is expected in December. 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.
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.
Merck KGaA and Pfizer are currently conducting a Phase II study to assess the safety and efficacy of avelumab in patients with metastatic MCC whose disease has progressed after at least one prior chemotherapy regimen.