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Medicine made free for extremely rare disease

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The Government has made a new medicine available for free for an extremely rare and life-threatening disease, potentially saving patients hundreds of thousands of dollars a year.

The new medicine Galafold® (migalastat) for the treatment of Fabry disease is now listed on the Life Saving Drugs Program, which provides free access to highly specialised medicines to treat patients with rare and life-threatening diseases.

Patients with Fabry disease have a rare enzyme deficiency, which means their bodies have trouble breaking down a fatty substance called globotriaosylceramide.

The condition usually presents in childhood with episodes of severe pain. Other symptoms include skins rashes, headaches, fatigue, vertigo, fever and vomiting and diarrhea.

It can result in potentially life-threatening complications including kidney failure, heart attack and stroke. It can have a major impact on patients and their families.

Currently, there are about 100 Fabry patients receiving enzyme replacement therapy through the LSDP.

Galafold® is a new oral medicine alternative for the treatment of Fabry disease patients aged 16 years and older.

It provides greater treatment choice for Fabry patients, reduces disease symptoms and dramatically improves quality of life, whilst also allowing patients to manage their own treatment at home without the need to have painful injections or infusions.

Without subsidy, Australian Fabry patients would pay hundreds of thousands of dollars for this treatment, putting them beyond the reach of most families who have to fight this extremely rare condition.

Health Minister Greg Hunt said it is the first medicine included on the Life Saving Drugs Program following the implementation of reforms to make the process more timely, transparent and improve patient access through the program.

“Our Government currently funds 14 different life-saving medicines for nine very rare diseases through the program, providing physical, emotional and financial relief for 400 Australian patients,” he said.

“These medicines are very expensive and would be too high of a financial burden on patients.”

Medicines funded through this program include high cost medicines that do not meet the criteria to be funded on the Pharmaceutical Benefits Scheme (PBS).

The new the Life Saving Drugs Program medical expert panel was announced in August. The panel, chaired by Australia’s former Deputy Chief Medical Officer Dr Tony Hobbs, supports the evaluation of medicines for funding on the program and provides advice to the Chief Medical Officer.