FDA NEWS RELEASE
For Immediate Release: Dec. 14, 2012
Media Inquiries: Stephanie Yao, 301-796-0394, stephanie.yao@fda.hhs.gov
Consumer Inquiries: 888-INFO-FDA
FDA approves Iclusig to treat two rare types of leukemia
Drug approved 3 months ahead of schedule
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 is being 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.
