ImmunoGen, Inc, a biotechnology company that develops targeted anticancer products using its antibody expertise and Targeted Antibody Payload (TAP) technology, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation to its IMGN901 compound when used for the treatment of Merkel cell carcinoma (MCC). Through a separate process, the European Union (EU) Committee for Orphan Medicinal Products (COMP) concurrently also granted IMGN901 orphan medicinal product designation for the treatment of MCC.

"IMGN901 has shown promising activity among the small number of MCC patients treated to date, suggesting this is a potential registration path for the compound," commented Daniel Junius, president and CEO. "Metastatic MCC is a rare and highly aggressive cancer with no approved treatments today. We are preparing to be ready to initiate pivotal testing of IMGN901 in MCC in 2011 and will make the final decision based on expanded clinical experience and regulatory input. Gaining orphan drug designation for IMGN901 in the US and Europe is an important step in this process."

Six patients with metastatic MCC had received IMGN901 at the time of the AACR-NCI-EORTC meeting in November 2009. Among these, two patients had an objective response - including a patient who has had a sustained complete remission - and a third patient had clinically meaningful stable disease.

In the US, the Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 people in this country. Orphan drug designation provides ImmunoGen with seven years of market exclusivity that begins once IMGN901 receives FDA marketing approval for the treatment of MCC. It also provides certain financial incentives that can help support the development of IMGN901 for MCC.

Similarly, in the EU, orphan medicinal product designation is intended to encourage companies to develop therapies for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU. Orphan medicinal product designation provides ImmunoGen with ten years of market exclusivity that begins once IMGN901 receives European approval for MCC. It also enables access to certain financial incentives as well as to protocol assistance.

IMGN901 is an investigational agent designed to kill cancer cells that express CD56, a protein.

MCC is an aggressive neuroendocrine cancer of the skin that typically occurs on the head/neck, most often in individuals of European ancestry.

ImmunoGen develops targeted anticancer therapeutics using its expertise in cancer biology, monoclonal antibodies and the creation and attachment of potent cell-killing agents.