RARE Daily

FDA Expands Approval of Chiesi’s Ferriprox for Transfusional Iron Overload due to Sickle Cell Disease

May 3, 2021

The U.S. Food and Drug Administration expanded the approval of Chiesi Global Rare Diseases’ Ferriprox for the treatment of patients with transfusional iron overload due to sickle cell disease or other anemias in adults and children age 3 and older.

Photo: Giacomo Chiesi, head of Chiesi Global Rare Diseases

This FDA approval expands the use of Ferriprox for patients with sickle cell disease or other anemias, as well as patients with thalassemia, regardless of prior iron chelation exposure.

Sickle cell disease (SCD) affects about 100,000 people in the United States and leads to a lower life expectancy by more than 20 years compared to the general population. SCD patients are typically diagnosed prior to 2 years of age and the more severe patients start to experience pain crises early in childhood. Many require hospitalization and chronic blood transfusions to manage disease complications. Disease complications include early onset stroke, acute chest syndrome, and multiorgan failure. Renal complications affect nearly 30-50 percent of patients with sickle cell anemia and account for 16-18 percent of mortality in adults with SCD.

Ferriprox is a synthetic, orally active iron-chelating agent shown to be effective in reducing iron concentration by penetrating cell membranes and removing toxic iron from organ tissues and extracellular fluids. Ferriprox is also FDA approved for the treatment of transfusional iron overload due to thalassemia syndromes, including beta-thalassemia, when current chelation therapy is inadequate.

“People who are living with SCD face significant challenges with pain and organ damage that can greatly impact their quality of life, and most who need blood transfusions also need iron chelation therapy including those with known kidney issues who have limited treatment options,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “We believe that delivering an iron chelation therapy that has no dosage adjustment required for patients with mild to severe renal impairment may address a significant unmet need in SCD.”

Author: Rare Daily Staff

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