Medicine for rare disease made free on Australia's Life Saving Drugs Program

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

The drug is Galafold (migalastat), from US rare disease focused biotech firm Amicus Therapeutics (Nasdaq: FOLD), and is for the treatment of Fabry disease. It will be listed on the Life Saving Drugs Program, which provides free access to highly specialized medicines to treat patients with rare and life-threatening diseases. Galafold was approved in Australia in August 2017 for the treatment of Fabry disease patients aged 16 years and older.

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

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. Others symptoms include skins rashes, headaches, fatigue, vertigo, fever and vomiting and diarrhea.

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

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.