FDA/CDC

FDA approves twice-daily formulation of key thalassemia drug


 

Chiesi Global Rare Diseases announced that the Food and Drug Administration has approved a new formulation of deferiprone (Ferriprox) for the treatment of patients with transfusional iron overload caused by thalassemia syndromes when current chelation therapy is inadequate. The new formulation of twice-a-day Ferriprox 1,000 mg oral tablets eliminates the midday dose, according to a company press release.

“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,” stated Thomas Coates, MD, in the press release. Dr. Coates is the section head of hematology at Children’s Hospital Los Angeles.

Deferiprone was originally approved by the FDA in 2011 for the treatment of transfusional iron overload caused by thalassemia syndromes. Ferriprox contains a label warning that it can cause agranulocytosis that can lead to serious infections and death. As neutropenia may precede the development of agranulocytosis, the warning advises measurement of the absolute neutrophil count before starting Ferriprox and monitoring the ANC weekly on therapy. In addition, Ferriprox should be interrupted if infection develops, and the ANC should be monitored more frequently.

As Ferriprox can cause fetal harm, women of reproductive potential should be advised to use an effective method of contraception during treatment and for at least 6 months after the last dose, according to the company release.

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