Deferiprone

Medical uses

Deferiprone monotherapy is indicated in the European Union for the treatment of iron overload in those with thalassaemia major when current chelation therapy is contraindicated or inadequate.

Deferiprone in combination with another chelator is indicated in the European Union in those with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload (mainly cardiac overload) justifies rapid or intensive correction.

The researchers found that the oral drug, deferiprone, reactivates the “altruistic suicide response” of an HIV-infected cell, killing the HIV RNA it carries. Effective suppression of HIV-1 generation and induction of apoptosis both require deferiprone at a concentration around 150 μM in infected T-cell lines. Since a 0.5 log10 decrement in HIV-1 RNA corresponds to an additional 2 years of AIDS-free survival and a 0.3 log10 decrement reduces the annual risk of progression to AIDS-related death by 25%, the measurements suggested biological significance.

Controversy

Deferiprone was at the center of a protracted struggle between Nancy Olivieri, a Canadian haematologist and researcher, and the Hospital for Sick Children and the pharmaceutical company Apotex, that started in 1996, and delayed approval of the drug in North America. Olivieri's data suggested that deferiprone can lead to progressive liver failure.

History

Deferiprone was approved for medical use in the European Union in August 1999.

It was approved for medical use in the United States in October 2011. Generic versions were approved in August 2019.

The safety and effectiveness of deferiprone is based on an analysis of data from twelve clinical studies in 236 participants. Participants in the study did not respond to prior iron chelation therapy. Deferiprone was considered a successful treatment for participants who experienced at least a 20 percent decrease in serum ferritin, a protein that stores iron in the body for later use. Half of the participants in the study experienced at least a 20 percent decrease in ferritin levels.

References

References

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6. (14 October 2011). "FDA Approves Ferripox (deferiprone) to Treat Patients with Excess Iron in the Body". U.S. [[Food and Drug Administration]] (FDA).
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9. Deepti Saxena, Michael Spino, Fernando Tricta, John Connelly, Bernadette M. Cracchiolo, Axel-Rainer Hanauske, Darlene D’Alliessi Gandolfi, Michael B. Mathews,Jonathan Karn,Bart Holland, Myung Hee Park, Tsafi Pe’ery, Paul E. Palumbo, Hartmut M. Hanauske-Abel (18 May 2016). [https://journals.plos.org/plosone/article?id=10.1371/journal.pone.0154842 Drug-Based Lead Discovery: The Novel Ablative Antiretroviral Profile of Deferiprone in HIV-1-Infected Cells and in HIV-Infected Treatment-Naive Subjects of a Double-Blind, Placebo-Controlled, Randomized Exploratory Trial].
10. (February 2004). "Introduction to The Olivieri symposium". Journal of Medical Ethics.
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13. Cribb, Robert. (27 February 2019). "UHN patients given unlicensed drug that led to diabetes, liver dysfunction and one death, study finds". The Star.
14. "Deferiprone: FDA-Approved Drugs".