New Leukemia Rx Hits Market

Iclusig approved to treat chronic myeloid leukemia and an acute lymphoblastic leukemia

(RxWiki News) Folks living with two types of blood cancer have new choices as of today. The US Food and Drug Administration has approved Iclusig (ponatinib) to treat chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). 

This drug operates differently than currently available therapies called tyrosine kinase inhibitors (TKI). In fact, this drug offers a new option to folks who have run out of TKI options.

"Ask your oncologist if you’re on the latest drug."

Iclusig gets goes after one target – a mutation called T3151 that promotes the growth of cancer. CML cells that have this mutation don’t respond to TKIs, which are the standard of care therapy. People with all phases of CML and resistant forms of Ph+ ALL take the new medication once a day.

"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, MD, director of the Office of Hematology and Oncology Products in the 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 the FDA’s commitment to approving safe and effective drugs for patients with rare diseases.”

The FDA approved Bosulif (bosutinib) in September, 2012, then 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.

The drug’s safety was evaluated in a clinical trial involving 449 patients with various phases of CML and Ph+ ALL, all of whom were treated with Iclusig.

The drug reduces the number of cells that have the Philadelphia chromosome mutation. That means people had what doctors call major cytogenetic response (MCyR).

Among the participants, 54 percent had major responses. Among patients with the T315I mutation, 70 percent achieved MCyR.

For folks who had more advanced cases of CML, the drug worked to normalize white blood cell counts or eliminate evidence of leukemia, which is called a major hematologic response or MaHR:

  • 54 percent of people with accelerated phase CML had a MaHR for a median 9.5 months.
  • 31 percent of patients with the earliest (blast) phase of CML saw a median 4.7 MaHR
  • 41 percent of people with Ph+ ALL had a median 3.2 MaHR.

The approval of Iclusig comes with a warning for health professionals and patients.The drug has been associated with blood clots and liver toxicity.

High blood pressure, rash, abdominal pain, fatigue, headache, dry skin, constipation, fever, joint pain and nausea were the most common side effects reported

This approval is three months ahead of schedule on the agency’s accelerated six-month review process. Action wasn’t expected until March 27, 2013.

Iclusig is marketed by ARIAD Pharmaceuticals. Bosulif is marketed by Pfizer, and Synribo is marketed by Teva Pharmaceuticals. Marqibo is marketed by Talon Therapeutics Inc.

Review Date: 
December 14, 2012