Officials with the FDA have approved deferiprone (Ferriprox, Chiesi Global Rare Diseases) twice-a-day tablets for the treatment of patients with transfusional iron overload due to thalassemia syndromes when current chelation therapy is inadequate.

The new formulation of twice-a-day deferiprone 1000 mg oral tablets eliminates the mid-day dose, and provides patients with the ability to reduce serum ferritin levels, and cardiac and liver iron, according to Chiesi.

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 with these conditions 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 inactivate iron by binding it in the tissues and circulation. Iron is then excreted from the body primarily via urine.

“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, Hematology at Children’s Hospital Los Angeles, in a prepared 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.”

Dosing is initiated at 75 mg/kg/day and can be increased up to 99 mg/kg/day to improve efficacy in iron chelation. The application for marketing approval was supported by bioequivalence studies, according to Chiesi.


REFERENCE

Chiesi Group Receives FDA Approval for Ferriprox® (deferiprone) twice-a-day tablets [news release]. Boston, MA; May 21, 2020: Chiei Global Rare Diseases [email]. Accessed May 21, 2020.