A MINDARIE family have welcomed the addition of a drug their son has trialled to the Life Saving Drug Program (LSDP).
The Federal Government recently announced that Vimizim will be available at no cost to Australian |patients who have Morquio A Syndrome from August 1.
Health Minister Greg Hunt said the treatment cost about $400,000 a year, putting it out of reach for many children with the disease.
“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,” he said.
“As a result they have |abnormal development and a possible early death.”
Sonia Antonio welcomed the news, as her son Coby (15) has the rare genetic condition and had been using the enzyme replacement therapy for almost four years, given to him compassionately by the manufacturer.
“We’re ecstatic, a huge weight has been lifted from our shoulders,” she said.
“Now we have peace of mind knowing he has this for life.
“His muscle tone, strength and stamina have all increased dramatically which makes him happier in himself.
“He’s also able to do more activities and keep up with his peers.
“There’s also all the internal benefits that we can’t see that’s going to give him such a better quality of life.”
Mrs Antonio said the listing meant other children with Morquio A would also have access to the drug.
“It’s the best news that these kids can start treatment at a young age,” she said.
“We have genuinely been overwhelmed by all the support for everyone who has backed the Morquio families in this fight.
“We encourage any other families to fight if they end up in the same situation.
“We never thought when we started our family that we would have to plead for medication for our son’s life but you just do it and keep fighting.”
Mr Hunt said Vimizim was only the 13th drug added to the LSDP.
“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,” he said. “It will cost $44 million over five years to treat 20 children.”
Manufacturer BioMarin said it was aware of 21 Australian patients living with Morquio A.
“The listing of Vimizim in Australia underscores the considerable effort made by the entire Morquio A, mucopolysaccharidosis and rare disease communities, who have worked tirelessly with BioMarin to ensure Australian patients could secure access to Vimizim,” BioMarin Australia manager Kathryn Evans said.