FDA Approves Deferiprone for Transfusional Iron Overload in Sickle Cell Patients

The US Food and Drug Administration (FDA) has approved deferiprone (Ferriprox) for the treatment of sickle cell or anemia-related transfusional iron overload in patients ≥3 years of age. This approval was granted to Chiesi Global Rare Diseases.

The synthetic, orally active iron-chelating agent, which is administered twice daily at 1000 mg, has been shown to effectively penetrate cell membranes and remove toxic iron from organ tissues and extracellular fluids.

As such, the FDA’s decision was based on a controlled study that showed that deferiprone was non-inferior to deferoxamine in inducing a change in liver iron concentration at 12 months from baseline. An extension study demonstrated a continued reduction in liver iron concentration over time—Mean concentration was 12.30 mg/g (from 14.93 mg/mg at baseline) at year 1, 11.19 mg/g at year 2, and 10.45 mg/g at year 3.

Associated adverse events were pyrexia, abdominal pain, bone pain, headache, vomiting, pain in extremity, sickle cell anemia with crisis, back pain, ALT increased, AST increased, arthralgia, oropharyngeal pain, nasopharyngitis, neutrophil count decreased, cough, and nausea.

"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, in a statement.

"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,” he continued. “We have a long history of commitment to the rare disease community and this FDA approval is a testament to the investments we continue to make in scientific research and development with patients at the center of everything we do.”

Deferiprone is also FDA approved for the treatment of transfusional iron overload due to thalassemias, including beta-thalassemia, patients ≥8 years of age.