Novartis says that its New Drug Application for Exjade (deferasirox) has been granted priority review by the US Food and Drug Administration as a once-daily oral iron chelator for the treatment of chronic iron overload due to blood transfusions. This means that the product could win the all-important green light in this market as early as November this year.
Iron overload is a potentially life-threatening condition that results from frequent blood transfusions required to treat certain types of anaemias and other disorders, such as sickle cell disease. If left undiagnosed or untreated, it can lead to damage of the liver, heart and endocrine glands, and can be fatal. Transfused patients are often treated for iron overload with a type of drug therapy called iron chelation, which removes excess iron from the body. The current standard of care is Desferal (deferoxamine), which is also manufactured by the Swiss company, but requires subcutaneous infusion lasting eight to twelve hours per day, for five to seven days a week for as long as the patient continues to receive blood transfusions, which could be life-long for many patients. Exjade, Novartis says, is taken once daily, after dispersing in a glass of water or orange juice.
Exjade was filed with the US, European, Swiss and Australian regulators in May 2005. The submissions were based on clinical trials involving more than 1,000 patients. A Phase III head-to-head trial with Desferal showed that Exjade significantly reduced liver iron concentration – an accepted indicator for body iron content.
It has been granted priority review in Australia and fast track status in Switzerland, and is designated as an orphan drug status in the US, Europe and Australia.