FDA Approves Deferiprone for Reducing Thalassemia-Related Iron Overload

The US Food and Drug Administration has approved a new twice-a-day tablet for the treatment of iron overload due to thalassemia syndromes.

Deferiprone (Ferriprox) received FDA approval on May 21 for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate, the Chiesi Group announced in a statement.

“In the management of patients with thalassemia, clinicians often see firsthand the difficulties they can have with dosing and compliance and the impact that this can have on the effectiveness of treatment,” said Thomas Coates, MD, section head of Hematology at Children’s Hospital Los Angeles, in a statement. “A treatment option that reduces serum ferritin, cardiac iron and liver iron with an established safety profile and now twice-a-day tablet dosing can represent a significant advantage for patients.”

Deferiprone is an iron chelator with the ability to reduce serum ferritin levels, which is common in patients with transfusional overload from thalassemia syndromes. Characterized by imap[ired production of hemoglobin, patients impact by the rare genetic disorder are at a greater risk of very high levels of iron in their blood and vital organs as a result of blood transfusion. Designed to bind to iron within the body’s tissues and circulation, deferiprone has the potential to help prevent unnecessary damage to the heart and liver.

According to prescribing information, dosing of deferiprone is initiated at 75 mg/kg/day and can be increased up to 99 mg/kg/day if needed. The statement from the Chiesi Group noted the approval of deferiprone is based on its ability to reduce serum ferritin levels and no trials have demonstrated a direct treatment benefit from use of the agent.

“The availability of a new twice-a-day oral tablet formulation of Ferriprox provides patients with the ability to reduce serum ferritin levels and cardiac and liver iron,” said Giacomo Chiesi, Head of Chiesi Global Rare Diseases, in the aforementioned statement. “This important milestone for Chiesi Global Rare Diseases is another reflection of our commitment to innovation to improve the lives of patients living with rare diseases around the world.”