A New Drug for Rare types of Leukemia

 A novel drug used to treat patients with chronic myeloid leukemia and Philadelphia chromosome positive acute leukemia has received approval from the U.S. Food and Drug Administration. The drug mostly works between leukemia patients who don't react to treatment from a class of drugs called tyrosine kinase inhibitors. It works by blocking proteins that cause cancerous cells to expand.

The drug name Iclusig is the third drug approved to treat CML and the second drug approved to treat ALL this year, demonstrating FDA's commitment to approving safe and effective drugs for patients with rare diseases. The approval of Iclusig is important because it provides a cure option to patients with CML who are not responding to other drugs, mainly those with the T315I mutation who have had few therapeutic options.

 The drug’s effectiveness was found in most CML patients, major cytogenetic response Fifty-four percent of all patients demonstrated by a reduction in the percentage of cells expressing the Philadelphia chromosome genetic mutation and 70 percent of patients with the T315I mutation achieved MCyR. The median extent of MCyR had not yet been reached at the time of analysis.

The approval of Iclusig is significant because it provides a treatment option to patients with CML who are not responding to other drugs, mainly those with the T315I mutation who have had few therapeutic options. Iclusig is the third drug approved to treat CML and the second drug accepted to treat ALL this year, demonstrating FDA's commitment to approving safe and effective drugs for patients with rare diseases.