FDA Approves new Leukemia Drug ahead of Time, Trials Successful

With no alternatives available for two rare types of leukemia, chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute leukemia (Ph+ ALL), the FDA has approved drug ahead of schedule.

In emphasizing the efficiency of the FDA in approving this drug among others, Richard Pazdur, director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, said, “The approval of Iclusig is important because it provides a treatment option to patients with CML who are not responding to other drugs, particularly those with the T315I mutation who have had few therapeutic options. 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.”

Leukemia patients are usually treated with tyrokine kinase inhibitors (TKIs) but in some cases, a mutation referred to as T315l resists this type of medication leaving them with no other form of therapy. This is why the approval of this drug, known as Iclusig and which is known to work with patients with these rare forms of leukemia, is vital.

In the testing phase, 449 patients with Ph+ ALL and CML who participated in these trials, found that this drug was extremely effective with major cytogenetic response occurring in 72 percent of those with the T315l mutation and 52 percent in all patients.

Some of the side effects include abdominal pain, headache, dry skin, high blood pressure, fever nausea and joint pain. Blood clots and liver toxicity is also possible when patient take these drugs, and patients will be informed of this risk by a warning label.