The FDA has 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. The drug “Iclusig” was reviewed by the FDA 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 those that are already in the market.

The drug is being approved by the federal agency 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. According to experts in the field of drug and pharmaceuticals the approval of the drug “Iclusig” is important in the sense that, it will offer a treatment option to patients who are suffering from CML and are not responding to existing drugs. The drug will be effective to those patients who are suffering from T315I mutation which is a rare disease and very few options are available to treat it.

While talking to industry experts and professionals the Director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, Richard Pazdur, says “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.”

Iclusig is one of those drugs that block synthesis and transportation of certain proteins that are critical to the development of carcinogenic or cancerous cells in the body. Iclusig is usually taken once a day to treat patients who are suffering from accelerated, chronic, and blast phases of CML and Ph+ ALL whose leukemia is resistant or intolerant to a class of drugs called tyrosine kinase inhibitors (TKIs). The drug was developed to target CML cells that have undergone T315I, a specific kind of mutation, known as T315I, which makes these cells resistant to currently approved TKIs.

The FDA has granted the drug a status of “Orphan Drug” which is usually given to those drugs that are intended to treat a rare disease or condition.

The drug Iclusig will be distributed and marketed with a Boxed Warning alerting health care professionals and patients about its potential risk to cause liver toxicity and create blood clots. Some of the side effects those were reported during the clinical trials of the drug are:

  • Abdominal pain
  • High blood pressure
  • Rash
  • Fatigue nausea
  • Joint pain
  • Headache
  • Dry skin
  • Constipation
  • Fever