Pharmion Corporation, along with its collaborator MethylGene Inc., today announced that the U.S. Food and Drug Administration (FDA) has designated the Companies' histone deacetylase (HDAC) inhibitor, MGCD0103, as an Orphan Drug for the treatment of Hodgkin's lymphoma in the United States.
Criteria for designation require that the product be intended for treatment of a condition affecting fewer than 200,000 people in the United States, and the application must include a rationale for the use of the drug in the rare disease or condition.
Preliminary data from a MGCD0103 Phase II monotherapy clinical trial in relapsed and refractory Hodgkin's lymphoma were presented at the American Society of Clinical Oncology 2007 Annual Meeting in June and were included in the application for Orphan Drug Designation. The data demonstrated an objective complete response (CR) plus partial response (PR) rate of 40 percent and a disease control rate (CR + PR + stable disease for > 6 cycles) of 45 percent in a population of 20 evaluable patients that had relapsed disease or were refractory (unresponsive) to all previous treatments, including, in most cases, bone marrow transplantation. Fatigue and gastrointestinal side effects were the most common adverse events and dose modification was effective in many of these patients.
Orphan Drug Designation allows special incentives for sponsors planning to test a product for use in a rare disease or condition. These incentives include, tax credits, research and development grant funding, and reduced filing fees during development or at the time of application for marketing approval. Once approved, the product may qualify for seven years of marketing exclusivity in the United States.
MGCD0103 is an orally-administered, isotype-selective HDAC inhibitor. The compound is currently in two Phase I/II clinical trials, in combination with Vidaza(R) for hematological malignancies and with Gemzar(R) in solid tumors, and in four Phase II monotherapy clinical trials in hematological malignancies.
Hodgkin's lymphoma (HL) is a cancer of the lymphatic system that begins in the lymph nodes and progresses to other organs, including the lungs, liver, bone and bone marrow. It is characterized by the presence of Reed-Sternberg cells. Currently, there is no known cause of the disease, but epigenetic alterations, including histone acetylation, have been identified. In addition, the Epstein-Barr virus, HIV and familial history are known risk factors. The disease is slightly more prevalent in men than women, and the median age of diagnosis is 38.
http://www.pharmion.com/ and http://www.methylgene.com/