Australia has admitted life-saving drug for Pompe disease in the Australian Life-Saving Drug Programme

Category: Archive
July 31, 2015

After years of hard work, persistence, and commitment, the Australian Pompe Association (APA) has achieved its goal–adult Pompe patients will finally be able to access treatment for Pompe disease beginning September 1, 2015.

Myozyme, a very expensive therapy, is the only treatment available for this life-threatening rare disease. On July 27, 2015, Sussan Ley, the Australian Minister for Health, approved Myozyme to be added to the Life Saving Drug Programme.

Pompe disease is a spectrum disease that can affect infants, children, and adults. Severity of the disease is usually determined by age of onset, but it can be fatal at any stage.

Myozyme has been available in many countries since 2006 after receiving approval by the regulatory authorities both in Europe (EMEA) and in the USA (FDA). It is the only approved therapy for Pompe disease.

In Australia, infantile-onset patients have been able to access treatment through the Life Saving Drug Programme since 2010; childhood onset patients, ages 2-18, have had access to treatment since February 2015. Now, adult Pompe patients will also be eligible for treatment starting in September 2015. Without Government subsidy through the Life Saving Drug Programme, Myozyme would be cost prohibitive to most Australians.

Helen Walker, a founding member of the APA and a board member of the International Pompe Association (IPA), initiated the "fight" to have treatment available for all Pompe patients in Australia. She was dedicated to this endeavour, but did not live to see the “fruits of her labor.” She died in 2012.

Continuing her legacy is the current president of the APA, Raymond Saich. He and other dedicated members of the APA continued to push this initiative forward, and they have finally managed to make it a reality. Now all Australian Pompe patients can receive this treatment as do patients in other countries throughout the world.

The IPA applauds the dedication and persistence of the APA. The determination of the Australian Pompe Association (APA) exemplifies the slogan of the internation Pompe community represented by the IPA: “Together We Are Strong.”

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