The drug Voxzogo hailed a breakthrough treatment for dwarfism

Dwarfism doesn’t just cause short stature, it can lead to sudden death. Now a new drug is seeing children hit growth targets.

The drug Voxzogo enables 10-year-old Oscar Rose, who has a passion for the Geelong Cats AFL team, to grow at the same rate as other children his age. Picture: Jay Town

Exclusive: 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”, was trialled in Australia and is set to revolutionise the way the condition is managed globally.

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

The 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 it $30 per script or $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 that 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 invasive surgery to deal with spinal cord compression and bowed legs – in some cases it can cause a type of seizure.

Professor Ravi Savarirayan with Oscar Rose. Picture: Supplied

The treatment is now being given to children from birth and there is early evidence it’s 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 were expecting to see that their growth curves are moving upwards and more towards the average, but we’ll need to get the real world evidence on that,” he said.

In Australia, around 140-160 children are born with the condition each year and there are around 400,000 people worldwide.

Children will not need the drug forever. The treatment will cease when skeleton stops growing and the growth plates fuse, which is about the age of 18.

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

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

The gene fault behind this form of dwarfism was discovered 30 years ago before US pharmaceutical company BioMarin Pharmaceutical discovered the potential treatment.

Danny Oscar and Hanna Rose with their son Oscar in February. Oscar has dwarfism.

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 apnoeic seizures that saw him hospitalised for most of the first 12 months of his life.

“I was breast feeding him at three months and he turned blue. A trip to the supermarket led to us performing CPR on our child at the checkout,” said his mother Hanna Rose.

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,” Ms Rose said, noting Oscar’s passion for AFL team the Geelong Cats.

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

“It’s not only in life changing but it is life saving, these complications can cause sudden infant death and paraplegia or problems with mobility.”

Originally published as The drug Voxzogo hailed a breakthrough treatment for dwarfism