The MDS Beacon™

The U.S. Food and Drug Administration announced on September 25 that it is examining the safety of Exjade (deferasirox) when prescribed to myelodysplastic syndromes (MDS) patients.  An initial FDA data analysis shows that MDS patients taking Exjade have a greater risk of serious side effects than patients without MDS.  The FDA announced it needs to further analyze the specifics of  the Exjade side effects, however, before coming to a final conclusion about the drug’s safety for MDS patients. 

Exjade is an iron chelating agent, a type of drug used to bind free iron in the blood and prevent it from forming deposits in organs.  It also works to remove excess iron that may have already built up in the heart, liver, and endocrine cells, and thus decreases the possibility of toxic effects.  Exjade is often prescribed to MDS patients who have excess iron, a condition often seen after repeated red blood cell transfusions.

The FDA data analysis showed that, compared to patients with other conditions, MDS patients were at higher risk for experiencing kidney failure, gastrointestinal bleeding, and death after taking Exjade.  However, the FDA has not yet made a conclusion about the drug’s safety because the adverse events may be  linked to several factors that often specifically characterize MDS patients, such as advanced age, the need for blood transfusions, and other medical complications.  The data analysis also suggested that  serious or fatal side effects were fewer in younger patients with conditions other than MDS that require blood transfusions. 

Novartis, the pharmaceutical company that markets Exjade, released the following statement in regard to the FDA investigation:

Patient safety is the highest priority at Novartis. As part of ongoing pharmacovigilance discussions with the FDA, we recently submitted a comprehensive safety analysis of various data sources regarding the use of Exjade for the treatment of transfusional iron overload in patients with myelodysplastic syndromes (MDS).  We have also shared this information with other regulatory authorities worldwide.

We are working with the FDA to further review and clarify the population of MDS patients most appropriate for treatment with Exjade and will update the label accordingly.  We take our obligations to patients and healthcare providers seriously and will work to ensure our label reflects the most up-to-date information to help physicians make optimal prescribing decisions.

Exjade continues to offer an important treatment option for appropriate patients with sickle cell disease, thalassemia, MDS and other rare anemias who are at risk for transfusional iron overload. Exjade has been used by more than 70,000 people worldwide.

While it reviews  Exjade’s safety, the FDA recommends patients speak to their own physicians for personalized treatment advice.  Physicians are still free to prescribe the drug to reduce excess iron in MDS patients.

Dr. Bart Scott, an MDS specialist at the University of Washington and Fred Hutchinson Cancer Research Center in Seattle, told the MDS Beacon that this new information about Exjade may slow its adoption among MDS patients.  He does not expect, however, the news to lead to a near-term drop in the number of MDS patients taking Exjade. 

Dr. Scott said that he personally would only use Exjade in patients who are transfusion dependent with ferritin levels greater than 1,000 and with a life expectancy greater than one year.

Deferoxamine (Desferal) is an alternative iron chelating drug available to MDS patients in the U.S.  The FDA also is currently reviewing an application by ApoPharma to market the iron chelation drug Ferriprox (deferiprone) in the U.S.  In addition, there is evidence that wheat grass juice may be a natural option for reducing excess iron.

For more information about the FDA’s announcement about Exjade’s safety, please see the FDA Web site.  The MDS Beacon also has a resource article with information about excess iron and chelation therapy in MDS patients.