Lorus drug gets FDA orphan status in leukemia

According to Lorus, this most recent orphan drug status designation in acute myeloid leukemia (AML), together with an ongoing clinical study program using GTI-2040 in different drug combinations, indicates potential for efficacy in a number of different tumor types. GTI-2040 was also granted orphan drug status for renal cell carcinoma in 2004.

The significance of orphan drug status is that it can result in the opportunity to obtain additional financial support from the US government for clinical study costs, exemption from certain fees at the time of submission of GTI-2040 to the FDA for marketing approval, and a grant of seven years of market exclusively in the US.

Orphan drug designation typically means that FDA marketing review times are expedited in comparison to other drugs since orphan drug status denotes serious or life-threatening diseases that afflict less than 200,000 patients annually in the US.

In partnership with the US National Cancer Institute’s cancer therapy evaluation program (CTEP) under a clinical trials agreement, GTI 2040 is being investigated in a phase II clinical trials program for AML, breast cancer, lung cancer, prostate cancer, colon cancer and a variety of solid tumors.