Health Canada OKs Using Ferriprox for Iron Overload

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by Patricia Inácio, PhD |

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Health Canada has expanded its approval of Chiesi Global Rare Diseases’ iron-binding oral treatment Ferriprox (deferiprone) to patients who have blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias.

The decision comes about five months after the U.S. Food and Drug Administration (FDA) approved Ferriprox for the same indication in the U.S. and one month after its approval in Brazil.

The therapy was approved in Canada previously for people with thalassemia major, another inherited blood disorder, who were experiencing iron overload when treatment with current iron-binding, or iron-chelating, agents was ineffective.

“We are very pleased that Health Canada recognizes and further validates the extensive clinical data and track record associated with Ferriprox to make this therapy available to more rare disease patients in the region,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in a press release.

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“This latest approval of Ferriprox in sickle cell disease represents our commitment to addressing the needs of underserved communities and providing patients and their families with options to address daily challenges as they navigate managing their disease,” said Fernando Tricta, head of Chiesi Canada.

People with SCD have difficulties in oxygen transport due to genetic mutations affecting the production of hemoglobin — the protein in red blood cells that carries oxygen through the body. As a result, patients often experience pain crises and organ damage, particularly in the kidneys.

Blood transfusions commonly are used as a way of delivering healthy red blood cells to patients with the goal of easing anemia. However, this can lead to an excessive buildup of iron in the body — a condition known as iron overload — that may damage internal organs.

Iron-chelating therapies bind to iron, helping the body to eliminate it, mainly through the urine. For that reason, these therapies are used in SCD patients receiving frequent blood transfusions.

“People living with sickle cell disease face debilitating symptoms including pain and organ damage, which often requires them to receive blood transfusions together with iron chelation therapy,” said Tricta.

Ferriprox is an oral iron-chelating agent that lowers iron levels by entering cells and removing toxic iron from organ tissues and fluids. It was developed originally by ApoPharma, which was acquired by Chiesi last year.

Previous data from a Phase 4 clinical trial called FIRST (NCT02041299) showed that Ferriprox was not inferior to Desferal (deferoxamine mesylate), another iron-chelating agent marketed by Novartis, at lowering iron levels in the liver of patients with SCD and other transfusion-dependent anemias after one year.

Also, liver iron levels continued to drop progressively for up to three years in patients given Ferriprox in the trial’s extension study (NCT02443545).

Among the most commonly reported adverse events in patients given Ferriprox were low neutrophil counts (neutropenia). Neutrophils are a group of immune cells that play a key role in defending the body against infections. A drop in their number may put patients at a higher risk of developing an infection.

Accordingly, Ferriprox’s label includes a warning regarding the possibility of severe neutropenia, as well as a recommendation for patients to monitor neutrophil levels every week while on the medication, to immediately report any symptoms that may indicate the presence of an infection, and to interrupt treatment if an infection arises.

“This important milestone is a testament to the hard work of our team, including the Canadian researchers who first developed Ferriprox, which was the first oral chelator approved in Canada. We are also grateful to the patients and clinicians who participated in our clinical research to make this achievement possible,” Chiesi added.