Providing life-saving drug to kids with rare disease

Providing life-saving drug to kids with rare disease

The Australian Government will provide a life-saving treatment to Australian patients who have a rare medical condition known as Morquio A Syndrome, at no cost.

Vimizim® (elosulfase alfa) will be available under the Life Saving Drugs Program from 1 August 2017.

With treatment costing around $400,000 each year, many Australian children suffering from Morquio A have been unable to receive treatment as it has simply been out-of-reach.

It’s an awful disease that cruelly affects children. Having access to Vimizim will be life-changing and indeed life-saving for some of our youngest Australians.

Morquio A Syndrome, or mucopolysaccharidosis (MPS) type IV A is an inherited metabolic condition, with Vimizim the only available treatment.

People born with the syndrome are either missing, or don’t have enough of, a crucial enzyme needed to break down long chains of sugar molecules. As a result they have abnormal development and a possible early death.

Before accessing this new drug, patients will need to undergo a clinical assessment and then yearly checks to ensure Vimizim treatment continues to be effective and appropriate for their condition.

Adding medicines to the Life Saving Drugs Program is rare and Vimizin is only the 13th medicine to be added.

It will cost of $44 million over five years to treat 20 children. We are able to make this life-changing listing because of our responsible financial management of the cost of medicines.

The Government has a rock solid commitment to Medicare and part of this commitment is ensuring people have access to medicine when they need it.

Source: Australian Government