Children with achondroplasia to benefit from life changing treatment added to PBS

The Murdoch Children’s Research Institute has welcomed the news that children born with the most common form of dwarfism, achondroplasia, will have access to a new life-changing treatment on the Pharmaceutical Benefits Scheme (PBS).

The Federal Government this week listed Voxzogo (vosoritide), manufactured by BioMarin, on the PBS for treatment of patients born with achondroplasia until final adult height is reached, at about 18 years of age.

Murdoch Children’s is the largest Voxzogo clinical trial site in the world, led by Professor Ravi Savarirayan. His research team has successfully shown, through ground-breaking clinical trials, how this drug that helps regulate bone development has boosted growth rates and functionality in children with achondroplasia.

Professor Savarirayan joined Federal Health Minister Mark Butler for the PBS announcement at Parliament House in Canberra on Wednesday.

Achondroplasia is the most common form of skeletal dysplasia or dwarfism, affecting around one in 20,000 babies born in Australia. It is caused by a genetic change (mutation), which results in impaired bone growth and disproportionately short limbs with numerous potential medical complications.

Professor Ravi Savarirayan speaks at the press conference with Federal Health Minister Mark ButlerProfessor Ravi Savarirayan speaks at Parliament House with Federal Health Minister Mark Butler.

Patients with achondroplasia are at risk of these complications throughout their lives and are 50 times more likely to die before the age of five than other children without the condition. Other complications include spinal cord compression, sleep apnoea, bowed legs, spinal stenosis and recurrent ear infections.

Voxzogo 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 this listing each year. The drug works directly on the growth plates of the bones, acting precisely on the underlying mechanism that causes the condition.

Professor Savarirayan said: “It’s a very proud day for our team (Molecular Therapies) at Murdoch Children’s, as we have played the pivotal role globally in the development of Voxzogo as a new treatment option for children with achondroplasia.

“As the first medicine available that targets the underlying cause of achondroplasia, Voxzogo results in increased bone growth in children with this condition and we believe this will translate to increased functionality, quality of life and decreased need for surgery in these children.

“The fact that this treatment is now on the PBS, and accessible to all Australian children with achondroplasia whose families choose to use it, is a landmark for the Australian achondroplasia community, the brave patients and their families who participated in our trials as well as my team here at the Murdoch Children’s.”

Kathryn North speaks at Parliament House with Professor Ravi Savarirayan and Federal Health Minister Mark ButlerProfessor Kathryn North speaks at Parliament House with Professor Ravi Savarirayan and Federal Health Minister Mark Butler

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Professor Ravi Savarirayan, Murdoch Children’s Group Leader, Molecular Therapies

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About Murdoch Children’s Research Institute

Murdoch Children's Research Institute is the largest child health research institute in Australia committed to making discoveries and developing treatments to improve child and adolescent health in Australia and around the world. They are pioneering new treatments, trialling better vaccines and improving ways of diagnosing and helping sick babies, children and adolescents. It is one of the only research institutes in Australia to offer genetic testing to find answers for families of children with previously undiagnosed conditions.