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Drug approved 3 months ahead of schedule

FDA approves Iclusig to treat two rare types of leukemia

Silver Spring, MD, USA (December 14, 2012) – The U.S. Food and Drug Administration today approved Iclusig (ponatinib) to treat adults with chronic myeloid leukemia (CML) and Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL), two rare blood and bone marrow diseases.

Iclusig is being approved more than three months ahead of the product’s prescription user fee goal date of March 27, 2013, the date the agency was scheduled to complete review of the drug application. The FDA reviewed the Iclusig drug application under the agency’s priority review program, which provides for an expedited six-month review for drugs that may provide safe and effective therapy when no satisfactory alternative therapy exists, or offer significant improvement compared to marketed products.

Iclusig blocks certain proteins that promote the development of cancerous cells. The drug is taken once a day to treat patients with chronic, accelerated, and blast phases of CML and Ph+ ALL whose leukemia is resistant or intolerant to a class of drugs called tyrosine kinase inhibitors (TKIs). Iclusig targets CML cells that have a particular mutation, known as T315I, which makes these cells resistant to currently approved TKIs.

“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,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research. “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 FDA approved Bosulif (bosutinib) in September 2012 and Synribo (omacetaxine mepesuccinate) in October 2012 to treat various phases of CML. Marqibo (vincristine sulfate liposome injection) was approved in August 2012 to treat Philadelphia chromosome negative ALL.

Iclusig is being approved under the agency’s accelerated approval program, which provides patients earlier access to promising new drugs while the company conducts additional studies to confirm the drug’s clinical benefit and safe use. The therapy was granted an orphan product designation because it is intended to treat a rare disease or condition.

Iclusig’s safety and effectiveness were evaluated in a single clinical trial of 449 patients with various phases of CML and Ph+ ALL. All participants were treated with Iclusig.

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

In accelerated and blast phase CML and Ph+ ALL, Iclusig’s effectiveness was determined by the number of patients who experienced a normalization of white blood cell counts or had no evidence of leukemia (major hematologic response or MaHR). Results showed:

• 52 percent of patients with accelerated phase CML experienced MaHR for a median duration of 9.5 months;
• 31 percent of patients with blast phase CML achieved MaHR for a median duration of 4.7 months; and
• 41 percent of patients with Ph+ ALL achieved MaHR for a median duration of 3.2 months.

Iclusig is being approved with a Boxed Warning alerting patients and health care professionals that the drug can cause blood clots and liver toxicity. The most common side effects reported during clinical trials include high blood pressure, rash, abdominal pain, fatigue, headache, dry skin, constipation, fever, joint pain, and nausea.

Iclusig is marketed by ARIAD Pharmaceuticals, based in Cambridge, Mass. Bosulif is marketed by New York City-based Pfizer, and Synribo is marketed by Frazer, Pa.-based Teva Pharmaceuticals. Marqibo is marketed by Talon Therapeutics Inc. based in South San Francisco, Calif.

For more information

• FDA: Office of Hematology and Oncology Products – http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm091745.htm
• FDA: Approved Drugs: Questions and Answers – http://www.fda.gov/Drugs/ResourcesForYou/Consumers/ucm054420.htm
• FDA: Drug Innovation – http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DrugInnovation/default.htm

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

U.S. Food and Drug Administration (FDA), 14.12.2012 (hB).