Deferiprone

Deferiprone (Ferriprox®) was first synthesized back in 1982 but it has been studied systematically only recently. Progress on drug development was curtailed in 1993 by Ciba-Geigy due to the toxic effects observed in non-clinical studies(mainly bone marrow suppression).

Nevertheless, the status of deferiprone was also reviewed at that time by clinical investigators from the International Study Group on Oral Iron Chelators, which clearly recommended the expansion of the clinical evaluation (Hershko, 1993). Since then, a number of additional studies have been undertaken on the drug, and its development has been re-instated by Apotex Holdings Inc (through its subsidiary ApoPharma) thanks to the effectiveness in chelation achieved in humans.

Deferiprone was authorised by the European Medicines Agency (Centralised Procedure) in August 1999 under “exceptional circumstances”. After presenting further data annually reviewed and on the basis of the encouraging results pertaining to the safety, in May 2002 the Committee for Medicinal Products for Human Use (CHMP) decided to remove the exceptional circumstances for the approval of deferiprone.

In 2004 an extension of indication was approved by the CHMP and the current deferiprone (Ferriprox®) indication has become: “Ferriprox is indicated for the treatment of iron overload in patients with thalassaemia major when deferoxamine therapy is contraindicated or inadequate” with the term ‘inadequate’ being interpreted as the use in patients who are non compliant to deferoxamine or when deferoxamine is not available. The supplementary protection certificate (SPC) has been updated specifically reporting: “There are limited data available on the use of deferiprone in children between 6 and 10 years of age, and no data on deferiprone use in children under 6 years of age”.