AUSTRALIA has joined more than 50 countries around the world in publicly funding an oral therapy for people with a potentially fatal condition known as Gaucher disease.
Sanofi's Cerdelga (eliglustat) will be added to Australia's Life Saving Drugs Program for the treatment of eligible adults with Gaucher disease type 1.
People with Gaucher disease do not have a sufficient level of an enzyme called beta-glucosidase (glucocerebrosidase) that breaks down a certain type of fat molecule.
This causes symptoms such as anaemia (low red blood cell counts), tiredness, easy bruising, an enlarged spleen and liver resulting in a protruding abdomen, bone pain and fractures.
The most common form of Gaucher disease, type 1, which affects 90% of people with the condition, generally does not affect the brain.
The Sanofi clinical development program for Cerdelga is the largest ever conducted in Gaucher disease, with approximately 400 patients treated in 29 countries.
Before starting treatment with Cerdelga, the patient is tested to see how rapidly their bodies break down the medicine.
Patients whose bodies break down Cerdelga very rapidly (ultrarapid metabolisers) and patients who have not been tested, or for whom results of the test are not clear, should not take this medicine.
Cerdelga can only be obtained with a prescription and treatment should be started and supervised by a doctor who has experience in the management of Gaucher disease.
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