Drug Approved Under Agency’s Orphan Drug and Accelerated Approval Programs
The FDA has approved Arranon (nelarabine) for marketing in the United States. This is a new drug to treat adults and children with T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL), whose disease has not responded to or has relapsed following at least two chemotherapy regimens. Arranon is the first drug to treat this limited population of patients.“With the approval of Arranon, the FDA has provided a significant option for those gravely ill populations that have no other proven effective alternatives,” said Dr. Steven Galson, Director of FDA’s Center for Drug Evaluation and Research (CDER). “Arranon has shown effectiveness in certain populations as a last-resort treatment, and we’re pleased to be able to approve it under our accelerated approval program.”
Arranon is a cancer chemotherapy drug that kills cancer cells by blocking the cell’s ability to reproduce. Rapidly dividing cancer cells are more sensitive to cancer chemotherapy drugs than are more slowly dividing normal cells.Arranon was approved under FDA’s accelerated approval program, which allows FDA to approve products for cancer and other serious or life-threatening diseases based on early evidence of a product’s effectiveness. In this case, this evidence consisted of complete disappearance of cancer cells in some patients, although in most cases the cancer later returned. In those patients who responded to Arranon, the disappearance of cancer cells was sometimes accompanied by return of normal blood cell counts. As required under FDA’s accelerated approval program, the sponsor will be completing further studies to verify Arranon’s clinical benefit.
Arranon also received Orphan Drug Designation, which is granted to products that treat rare diseases (those that affect fewer than 200,000 people in the United States). The Orphan Drug Act provides a seven-year period of exclusive U.S. marketing to the first sponsor that obtains marketing approval for a designated orphan drug. Approximately 1600 patients are newly diagnosed with T-ALL/T-LBL each year in the United States. Of those, an estimated 500 patients per year have relapsed or refractory T-ALL/T-LBL, with approximately 200 of these patients being children.
Acute lymphoblastic leukemia (ALL) is an aggressive disease that progresses rapidly without effective therapy. Each year, an estimated 2400 children and 1200 adults are diagnosed with ALL in the U.S., of whom, approximately 700 have T-ALL. Lymphoblastic lymphoma (LBL) represents approximately 30% of childhood and 3% of adult non-Hodgkin’s lymphoma (NHL). An estimated 50,000 patients are diagnosed with NHL each year, of whom, approximately 900 have T-LBL.
