FDA approves Ferriprox for transfusional iron overload due to sickle cell disease
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The FDA approved deferiprone for treatment of children or adults with transfusional iron overload due to sickle cell disease or other anemias.
The indication applies to use of the agent for patients aged 3 years or older.
Deferiprone (Ferriprox, Chiesi Farmaceutici) is a synthetic, orally active iron-chelating agent designed to reduce iron concentration by penetrating cell membranes and removing toxic iron from organ tissues and extracellular fluids.
"People who are living with sickle cell disease face significant challenges with pain and organ damage that can greatly impact their quality of life,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in a company-issued press release. “Most who need blood transfusions also need iron chelation therapy, including those with known kidney issues who have limited treatment options. 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 sickle cell disease.”
The FDA based the approval on results of a study that compared deferiprone with deferoxamine for patients with sickle cell disease or other transfusion-dependent anemias. The study met the noninferiority criterion for change in liver concentration after 12 months, according to the release.
Results of an extension study showed liver iron concentrations continued to decline over time after deferiprone treatment, from 14.93 mg/g dry weight at baseline to 12.3 mg/g dry weight after 1 year, 11.19 mg/g dry weight after 2 years and 10.45 mg/g dry weight after 3 years.
The most common adverse events reported among patients treated with deferiprone in clinical trials included pyrexia, abdominal pain, bone pain, headache, vomiting, extremity pain, sickle cell anemia with crisis, back pain, increased alanine aminotransferase and aspartate aminotransferase levels, arthralgia, oropharyngeal pain, nasopharyngitis, decreased neutrophil count, cough and nausea.
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