Summary Safety Review - Ferriprox (deferiprone) - Assessing the potential risk of brain and nervous system (neurological) disorders in children

Review decision

A Summary Safety Review complements other safety related information to help Canadians make informed decisions about their use of health products. Each summary outlines what was assessed in Health Canada’s review, what was found and what action was taken by Health Canada, if any.


Issued: 2019-02-25

Product

Ferriprox (deferiprone)

Potential Safety Issue

Brain and nervous system (neurological) disorders

Key Messages

  • Ferriprox (deferiprone) is a prescription drug authorized for sale in Canada to remove excess iron accumulated in the body from blood transfusions in the treatment of thalassemia syndromes (genetic diseases of blood production). Ferriprox is recommended for use when other iron removal therapies do not work well enough.
  • Health Canada assessed the potential risk of brain and nervous system (neurological) disorders, such as difficulty walking or difficulty with the coordination of movement in children treated with Ferriprox at recommended doses. Health Canada's review was triggered by 2 published cases1, 2.
  • The product safety information for Ferriprox already warns of this risk when children are treated with 2.5 times the recommended dose of Ferriprox.
  • Health Canada's review concluded that there may be a link between Ferriprox and the risk of neurological disorders in children treated at recommended doses. Health Canada will notify the manufacturer to update the safety information for Ferriprox to warn that cases of neurological disorders in children have also been observed at recommended doses of Ferriprox.

Overview

Health Canada reviewed the potential risk of neurological disorders such as difficulty walking or difficulty with the coordination of movement in children when Ferriprox is used at recommended doses. This safety review was triggered after 2 reports1, 2 were published suggesting this risk in children taking Ferriprox. At the time of the review, the product safety information for Ferriprox included a warning to inform that similar neurological disorders have been observed in 2 children treated with 2.5 times the recommended doses of Ferriprox.

Use in Canada

  • Ferriprox (deferiprone) is a prescription drug authorized for sale in Canada for the treatment of patients with excess accumulated iron from repeated blood transfusions as part of the treatment of thalassemia syndromes (genetic diseases of blood production). Ferriprox is recommended for use when other iron removal (i.e. chelation) therapies do not work well enough.
  • Deferiprone has been marketed in Canada since July 2016 under the brand name Ferriprox. This drug is available only through a controlled distribution program called Ferriprox Assist.

Safety Review Findings

  • At the time of the review, Health Canada had not received any Canadian reportsa of neurological disorders linked to the use of Ferriprox in children.
  • Health Canada's safety review found 2 international casereports1, 2 of neurological disorders in children that were treated with Ferriprox at recommended doses. In one report, there was a link between neurological disorders and the use of Ferriprox. In the other report, the link between neurological disorders and the use of Ferriprox was possible, but there were other factors (such as, the treatment condition, and other neurological/medical conditions) that may have also caused the neurological disorders.
  • In 2017, the European Medicines Agency (EMA) included the risk of neurological disorders in children when exposed at the recommended doses of deferiprone in the product safety information for Ferriprox. This risk of neurological disorders at recommended doses is not labelled in the United States.

Conclusions and actions

  • Health Canada's review concluded that there may be a link between Ferriprox and neurological disorders in children when it is used at recommended doses, and not only at higher doses as currently referenced in the product safety information.
  • Health Canada will notify the manufacturer to update the current warnings in the safety information to reflect that cases of neurological disorders in children have also been observed at recommended doses of Ferriprox. Health Canada encourages consumers and healthcare professionals to report any side effects related to the use of this or other health products.
  • Health Canada will continue to monitor the safety information involving Ferriprox, as it does for all health products on the Canadian market, to identify and assess potential harms. Health Canada will take appropriate and timely action if or when new health risks are identified.

Additional information

The analysis that contributed to this safety review included scientific and medical literature, international adverse reaction reports, and what is known about the use of this drug both in Canada and internationally.

For additional information, contact the Marketed Health Products Directorate.

References

  1. Economou M, Teli A, Gourtsa V, Vargiami E, Zafeiriou D. Neurological Involvement in a Thalassemic Child Receiving Deferiprone. J Pediatr Hematol Oncol 2016;38(5):406.
  2. Parakh N, Sharma R, Prakash O et al. Neurological Complications and Cataract in a Child With Thalassemia Major Treated With Deferiprone. J Pediatr Hematol Oncol 2015;37(7):e433-e434.

Footnotes

  1. Canadian reports can be accessed through the Canada Vigilance Online Database.

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