May 1, 2021
The U.S. FDA has approved a new indication for Ferriprox® (deferiprone), manufactured by Chiesi USA, to treat transfusional iron overload due to sickle cell disease (SCD) or other anemias in patients who are at least three years old. In addition, Ferriprox’s indication to treat transfusional iron overload due to thalassemia syndromes has been expanded beyond adults to include pediatric patients who are at least three years of age.
Patients who have SCD, anemia, or thalassemia may require blood transfusions as part of treatment. However, this can lead to the body absorbing excess levels of iron. The extra iron may accumulate in organs such as the liver and heart with potentially life-threatening consequences. Ferriprox works by attaching to iron in the body and helping to remove it through the urinary tract.
In clinical trials, Ferriprox demonstrated non-inferiority to deferoxamine (a current standard of care) in treating transfusional iron overload due to SCD. In addition, liver iron concentrations continued to decrease over time with Ferriprox therapy in trial participants.
Recommended dosing is based on the patient’s body weight. Ferriprox oral solution is indicated for use in patients three years and older, while Ferriprox oral tablets are indicated only for patients who are least eight years old. A black box warning cautions that Ferriprox can cause agranulocytosis (a severe form of low white blood cell count), which can lead to serious infections and death and may be preceded by neutropenia (a low count of a specific type of white blood cell called neutrophils). Patients on Ferriprox should have their absolute neutrophil count monitored on a weekly basis throughout therapy.
Ferriprox first received FDA approval in 2011.