New Drug Treats Dwarfism

Life-changing breakthrough for children

Murdoch Children’s Research Institute (MCRI) recently shared a new article from the Herald Sun titled ‘New Drug Treats Dwarfism’ written by Sue Dunlevy. In the post, MCRI shared this:

Murdoch Children’s Professor Ravi Savarirayan has been on the cutting-edge of treatment for Achondroplasia, with a massive win secured this week for children with the bone disorder.

Federal Health Minister Mark Butler has announced that new bone growth treatment Voxzogo has been listed on the PBS and now costs only $30 per script. Before the subsidy, families were paying up to $330,000 per year.

Professor Savarirayan led key research to help to develop this treatment, which has now become highly accessible.

Murdoch Children’s Research Institute Facebook page

Children born with dwarfism could overcome their short stature if treated with a new life-altering drug.

The treatment, which “allows average bone growth to resume,” has been tested in Australia and is set to revolutionize the way the condition is managed globally.”

The drug Voxzogo is delivered as a daily injection in a cartridge similar to an insulin shot. It targets the underlying cause of the genetic condition Achondroplasia, the most common form of dwarfism.

A worldwide trial of the treatment, led by Murdoch Children’s Research Institute’s Professor Ravi Savarirayan, was so successful it has just received an Australian government subsidy – making $30 per script of $7.30 with a concession card. Before the subsidy, the drug cost more than $330,000 per year.

“In this form of dwarfism, it’s like trying to drive your car with your handbrake on. It can’t reach average speeds and so the bones can’t grow normally,” Professor Savarirayan said.

“This medication releases the handbrake from a molecular point of view, and allows average bone growth to resume.”

Not only do people with the condition have short stature, children with the condition are 50 times more likely to suffer sudden death in the first five years of life.

They often require surgery to deal with spinal cord compression and bowed legs.

The treatment is now being given to children from birth. There was early evidence ut was relieving common complications that require surgery to correct growth problems at the base of the skull and compression of the spinal cord, Professor Savarirayan said.

“If the child gets the drug from very early on, we are expecting to see that their growth curves are moving upwards and more towards the average, but we’ll need to get the evidence on that,” he said.

In Australian about 150 children are born with the condition each year. The treatment will cease when the skeleton stops growing and growth plates fuse, which is around the age of 18.

“Listing Voxzogo on the Pharmaceutical Benefits Scheme could be life-changing for children born with dwarfism and their families,” Health Minister Mark Butler said.

“It is the first time effective treatment will be affordable for every family affected by this genetic condition.”

Ten year old Oscar Rose had major surgery to remove two vertebrae from his spine and relieve pressure from his brain stem at age three months.

His Achondroplasia also caused a rare form of recurrent seizures that saw him hospitalised for most of the first 12 months of his life.

Enrolled in the Voxzogo trial at the age of five, Oscar is now growing at the same rate as other children his age.

“He is absolutely so active. We can’t stop him. He’s up early And he wants to go all day is kick the footy,” said his mum Hanna Rose.

“We just feel so overwhelmed and so grateful to live in this time and have access to this medication – and for the doctors who have worked on it for 30 years.”