Chiesi Group gets FDA blessing for new formulation of Ferriprox

Patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate have a new treatment option. The Food and Drug Administration has given the green light to Chiesi Group’s Ferriprox (deferiprone) in twice-a-day 1,000 mg oral tablets that eliminates the mid-day dose.

“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 “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.”

Thalassemia syndromes are a group of rare inherited hematological conditions including beta-thalassemia and are characterized by impaired hemoglobin production. In certain cases, such as with beta-thalassemia, severe forms of this genetic disorder if left untreated can lead to life-threatening complications. Patients are often managed with long-term blood transfusions that can put them at risk of developing very high levels of iron in their blood and vital organs. As the level of iron rises, it can generate free radicals that can be toxic to proteins and membranes. Deferiprone is engineered to bind to iron in the tissues and circulation, thereby inactivating it. Iron is then excreted from the body primarily via urine. Dosing is initiated at 75 mg/kg/day and can be increased up to 99 mg/kg/day to improve efficacy in iron chelation. 

“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,  section head, Hematology at Children’s Hospital Los Angeles. “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.”