Ferriprox Approved for Transfusional Iron Overload Due to Sickle Cell Disease

The Food and Drug Administration (FDA) has expanded the approval of Ferriprox^® (deferiprone) tablets to include the treatment of transfusional iron overload due to sickle cell disease (SCD) or other anemias in patients 8 years of age and older. Additionally, the oral solution formulation of Ferriprox has been approved for use in patients 3 years of age and older for this indication, as well as in patients with thalassemia syndromes.

Ferriprox is an oral chelating agent that binds with ferric ions (iron III) to form neutral 3:1 (deferiprone:iron) complexes. The approval was based on data from an active-controlled noninferiority study (ClinicalTrials.gov: NCT02041299) that compared the efficacy of Ferriprox to deferoxamine in 185 patients with SCD and other transfusion-dependent anemias by evaluating liver iron concentration (LIC). Patients were randomly assigned 2:1 to receive either Ferriprox (n=122) or deferoxamine (n=63).

Results showed that Ferriprox met the noninferiority criterion for change in LIC from baseline after 12 months (primary endpoint); the least squares estimate of mean decrease from baseline in LIC was 4.13 ± 0.50mg/g dry weight (dw) for Ferriprox compared with 4.38 ± 0.59mg/g dw for deferoxamine. Data from an extension study demonstrated that the LIC continued to decrease over time in Ferriprox-treated patients. The mean LIC value decreased from 14.93mg/g dw at baseline to 12.30mg/g dw after 1 year of treatment, to 11.19mg/g dw after 2 years, and to 10.45mg/g dw after 3 years. 

As for safety, the most common adverse reactions reported in patients with SCD or other anemias were pyrexia, abdominal and back pain, bone pain, headache, vomiting, pain in extremity, sickle cell anemia with crisis, increased ALT/AST, arthralgia, oropharyngeal pain, nasopharyngitis, decreased neutrophil count, cough, and nausea.

Ferriprox is supplied as 500mg tablets in a 100-count bottle, 1000mg tablets (twice a day presentation) in a 50- and 500-count bottle, 1000mg tablets (three times a day presentation) in a 50-count bottle, and 100mg/mL oral solution in a 500mL bottle.

References

1. Chiesi Global Rare Diseases announces FDA approval of Ferriprox^® (deferiprone) for treatment of transfusional iron overload due to sickle cell disease. [press release]. Boston, MA: Chiesi Global Rare Diseases; May 1, 2021. 
2. Ferriprox [package insert]. Cary, NC: Chiesi USA, Inc.; 2021.