Children born with the most common form of dwarfism will have access to a new life changing treatment on the Pharmaceutical Benefits Scheme (PBS).
The Australian Government will list Voxzogo® (vosoritide) on the PBS for treatment of patients with achondroplasia whose growth plates are not yet closed.
Achondroplasia is the most common form of skeletal dysplasia or dwarfism, affecting around one in 20,000 babies born in Australia.
It is a rare condition caused by a genetic mutation which results in impaired bone growth and disproportionately short limbs.
This is the first and only approved medicine on the PBS that targets the underlying cause of achondroplasia. Around 140 children are expected to benefit from the listing each year.
Voxzogo works directly on the growth points of the bones to promote new bone growth in patients whose bones are still growing.
Patients with achondroplasia are also at risk of severe complications throughout their lives and are almost 50 times more likely to die before the age of 5 than other children.
Australia has played a leading role in the development of Voxzogo - with the Murdoch Children's Research Institute at the Royal Children's Hospital in Melbourne becoming the largest Voxzogo clinical trial site in the world, led by Australian Clinical Geneticist and world-renowned achondroplasia expert Professor Ravi Savarirayan.
Without subsidy, Voxzogo could cost more than $330,000 per year. Through the PBS, Australian families with eligible children will pay a maximum of $30 per script or just $7.30 with a concession card.
Quotes Attributable to Minister Butler:
"Listing Voxzogo on the PBS could be life changing for children born with dwarfism and their families.
"It is the first time an effective treatment will be affordable for every family with an eligible child affected by this genetic condition.
"I want to particularly congratulate the work of the Murdoch Children's Research Institute and Professor Ravi Savarirayan who led BioMarin's development program for Voxzogo, with the largest clinical trial site in the world."
