FDA approves expanded use for Chiesi’s sickle cell drug Ferriprox

Italy’s Chiesi Global Rare Diseases has gained approval from the US Food and Drug Administration (FDA) for the expanded use of its sickle cell disease (SCD) treatment Ferriprox.

Ferriprox (deferiprone) is now approved in the US for the treatment of transfusional iron overload due to SCD or other anaemias in adult and paediatric patients aged three years or older.

The new approval expands the use of Ferriprox beyond its other indication, where it is approved to treat patients with transfusional iron overload due to thalassemia syndromes.

In the US, SCD affects approximately 100,000 people and is associated with a lower life expectancy of over 20 years compared with the general population.

Ferriprox, a synthetic and orally active iron-chelating agent, is designed to penetrate cell membranes and remove toxic iron from organ tissues and extracellular fluids.

In a study comparing the efficacy of Ferriprox to deferoxamine in patients with SCD and other transfusion-dependent anaemias, Chiesi’s drug met the non-inferiority criterion for change in liver iron concentration from baseline after 12 months.

An extension study also confirmed that liver iron centration continued to decrease progressively over time after three years of treatment with Ferriprox, according to Chiesi.

In clinical trials of Ferriprox in SCD patients, the most commonly reported adverse reactions included fever, abdominal pain, bone pain, headache, vomiting, pain in extremity, sickle cell anaemia with crisis, back pain, ALT increase, AST increase, 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.

"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. 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 centre of everything we do,” he added.