MGI Pharma’s Dacogen gets US orphan status in leukemia

Dacogen (decitabine) is a hypomethylating agent believed to exert its antineoplastic effects by incorporation into DNA and inhibition of an enzyme called DNA methyltransferase. Dacogen-induced hypomethylation in neoplastic cells may restore normal function to genes that are critical for the control of cellular differentiation and proliferation.

Dacogen was approved by the FDA on May 2, 2006 for the treatment of patients with myelodysplastic syndromes (MDS) including previously treated and untreated, de novo and secondary MDS of all French-American-British (FAB) subtypes, and intermediate-1, intermediate-2, and high-risk international prognostic scoring system (IPSS) groups.

Orphan drug designation is designed to promote the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people per year in the US. The designation may provide seven years of market exclusivity for Dacogen in the acute myeloid leukemia (AML) indication following FDA approval.

Dacogen is being co-developed by MGI Pharma and Janssen-Cilag, a Johnson & Johnson company. Janssen-Cilag companies are responsible for regulatory and commercial activities in all territories outside North America, while MGI Pharma retains responsibility for all activities in the US, Canada and Mexico.

MGI Pharma is currently conducting a phase III pivotal trial to evaluate Dacogen in patients with AML. Additional phase II studies are also underway to evaluate alternative dosing regimens for Dacogen in patients with MDS, AML and chronic myelogenous leukemia.