Ferriprox Approved in Brazil to Treat Iron Overload in SCD Patients

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by Marta Figueiredo PhD |

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Regulators in Brazil have approved Ferriprox (deferiprone), Chiesi Global Rare Diseases’ iron-binding oral treatment, for blood transfusion-induced iron overload in people with sickle cell disease (SCD) or other anemias.

Ferriprox’s use was also approved in combination with other iron-binding, or iron-chelating, treatments for people with thalassemia major, a severe inherited blood disorder, when single therapy is ineffective, or when the consequences of iron overload are seen as life threatening.

The therapy was previously available in the country to treat transfusional iron overload in thalassemia major patients when current chelation therapy was inadequate and not recommended.

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“We are pleased to announce these new indications for Ferriprox in Brazil in sickle cell disease and as a combination therapy in thalassemia major, which marks the first time in 15 years that an iron chelation therapy has been approved in the country,” Giacomo Chiesi, head of the company, said in a press release.

Carlos Chueiri, the medical director of Chiesi Brazil, said that the approval “is a meaningful achievement that will benefit a significant portion of patients affected by sickle cell disease, which is the most common hereditary anemia in Brazil.”

The decision by Brazil’s National Health Surveillance Agency follows a similar label expansion in the U.S. — where the therapy was available for thalassemia patients when current iron-chelating medication was ineffective — to include adults and children, ages 3 and older, with SCD or other anemias.

Blood transfusions, which deliver normal red blood cells to patients, are commonly used to help lessen anemia in people with SCD, thalassemia, and other anemia-related conditions. In SCD patients, blood transfusions also help to lower blood viscosity (thickness and stickiness), reducing pain crises and organ damage.

Transfusions, however, can lead to complications like iron overload — a condition in which there is an excessive buildup of iron in the body, which can damage organs such as the heart and liver.

Ferriprox is a lab-made, orally available iron-chelating agent that lowers iron levels by entering cells and removing toxic iron from organ tissues and fluids. By helping to remove excess iron, Ferriprox has the potential to ease iron overload and prevent its damaging effects.

Ferriprox’s safety and effectiveness were compared with those of Novartis’ iron-chelating agent Desferal (deferoxamine mesylate) in 230 people, ages 2 and older, with SCD and other transfusion-dependent anemias in a Phase 4 trial, called FIRST (NCT02041299).

Results showed that Ferriprox was not inferior to standard Desferal, given through under-the-skin injections, at reducing iron levels in the liver after one year of treatment, Chiesi reported in May.

In addition, FIRST’s extension study (NCT02443545), in which all patients received Ferriprox, confirmed that liver iron levels continued to drop progressively up to three years.

The most commonly reported adverse events during Ferriprox clinical trials in people with SCD or other anemias included fever, headache, vomiting, nausea, abdominal and extremity pain, pain crises, higher-than-normal levels of liver enzymes (suggestive of liver damage and inflammation), and low neutrophil counts (neutropenia).

A severe drop in neutrophils, an immune cell involved in fighting against infections, can increase the risk of an infection.

Ferriprox’s label includes a warning regarding the possibility of severe neutropenia, as well as a recommendation for monitoring neutrophil levels weekly while on the medication and to interrupt treatment in the presence of an infection.

The medication was originally developed by ApoPharma, and acquired by Chiesi in 2020.