Children Benefit from New Drug on Life Saving Drugs Program
The Australian Government will provide $4.2 million over four years to fully subsidise Myozyme® (alglucosidase alpha) to treat the rare and serious enzyme deficiency disorder, Infantile-onset Pompe disease
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21 February 2010
The Australian Government will provide $4.2 million over four years to fully subsidise Myozyme® (alglucosidase alpha) to treat the rare and serious enzyme deficiency disorder, Infantile-onset Pompe disease.
It is estimated between three and eleven patients with the disease will be treated through the Life Saving Drugs Program over the four years. The average cost in the first year of therapy is estimated at $109,600 per person. This will be fully subsidised by the Government for eligible patients for the first time as of this month.
Infantile-onset Pompe disease patients are deficient in an enzyme (acid alpha-glucosidase) required for the breakdown of glycogen (a form of starch) in the cells of the body. The steady accumulation of glycogen in the body's tissues (in particular the heart muscle) leads to progressive debility, organ failure and death.
Myozyme is an enzyme replacement therapy which breaks down accumulated glycogen and therefore halts the progression of Infantile-onset Pompe disease.
The Government provides funds for the purpose of assisting access to expensive and lifesaving drugs accepted by the Pharmaceutical Benefits Advisory Committee (PBAC) as clinically effective, but not available as pharmaceutical benefits because of a failure to meet cost effectiveness criteria.
Eligibility for a patient’s treatment is determined against guidelines and the advice and recommendations of an expert advisory committee, the Infantile-onset Pompe Disease Advisory Committee.
Due to the severe and rapid progression of Infantile-onset Pompe disease there is an urgency to commence treatment with Myozyme in order for it to be effective. The Committee urges parents and physicians to make an application for treatment with Myozyme promptly after diagnosis.
In 2008-09 Government expenditure under the Life Saving Drugs Program was approximately $41 million.
For all media inquiries, please contact the Minister's Office on 02 6277 7220
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