Extended-release formulation of Ferriprox now approved in Canada

Health Canada has approved an extended-release formulation of Ferriprox (deferiprone) to treat iron overload in people with sickle cell disease (SCD) and other blood disorders.

The key benefit of the new formulation, according to the therapy’s maker, Chiesi Global Rare Diseases, is that it reduces the medication’s dosing frequency: While the original formulation of Ferriprox needs to be taken three times a day, the extended-release version is taken twice daily instead.

“As a family business and a B Corp focused on the long-term support of rare disease communities, we have the opportunity to remain close to the patients, caregivers and their providers, always learning from them and responding to their needs,” Giacomo Chiesi, head of Chiesi Global Rare Diseases, said in a company press release.

“That is why we found it important to pursue the development of a twice-a-day tablet formulation of Ferriprox,” Chiesi said.

Treatment of blood diseases like SCD often involves blood transfusions to help maintain a healthy supply of blood cells in the body. However, because blood is rich in iron, frequent blood transfusions can lead to iron overload — excessive levels of iron in the body that may damage internal organs.

Ferriprox is an oral iron-chelating agent — a medication designed to bind to iron and help clear it from the body, thereby lowering its levels. It was originally developed by ApoPharma, and acquired in 2020 by Chiesi, which is based in Parma, Italy.

The original formulation of Ferriprox was approved in Canada in 2015 as a treatment for iron overload when other iron-chelating therapies are inadequate. It is indicated for thalassemia syndromes, which are a group of genetic blood disorders that cause red blood cells to have lower than normal levels of the oxygen-binding protein hemoglobin.

Health Canada expanded the approval of Ferriprox in 2021 to cover the treatment of iron overload due to SCD and other anemias. The therapy also received similar approvals in the U.S. and Brazil that year.

Approvals of Ferriprox in SCD were supported by data from a Phase 4 clinical trial called FIRST (NCT02041299), as well as its two-year extension study, FIRST-EXT (NCT02443545).

The FIRST trial showed that Ferriprox was not inferior to Desferal (deferoxamine mesylate) — another approved iron-lowering medication marketed by Novartis. Subsequent findings from the extension study showed that long-term treatment with Ferriprox was generally safe and effective for reducing iron levels.