New life-changing treatment for dwarfism added to PBS


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Children born with the most common form of dwarfism have access to new, life-changing treatments through the Pharmaceutical Benefits System (PBS).

The Australian government will list Voxzogo® (vosolitide) on PBS for the treatment of achondroplasia patients whose growth plates have not yet closed.

Achondroplasia is the most common form of skeletal dysplasia or dwarfism, affecting approximately 1 in 20,000 babies born in Australia.

It is a rare disease caused by a genetic mutation that causes impaired bone growth and disproportionately short limbs.

It is the first and only approved drug in PBS to target the underlying cause of achondroplasia. About 140 children are expected to benefit from this list each year.

Voxzogo works directly on the bone growth point to promote new bone growth in patients whose bone is still growing.

People with achondroplasia are at risk of serious complications throughout their lives and are almost 50 times more likely to die before age 5 than other children.

Australia has played a leading role in the development of Voxzogo, with the Murdoch Institute for Pediatrics at the Royal Children’s Hospital Melbourne, led by Professor Ravi Savarayan, an Australian clinical geneticist and world-renowned expert in achondroplasia. It is the largest Voxzogo clinical trial facility in the world. .

Without subsidies, Voxzogo could cost over $330,000 a year. Through PBS, Australian families with eligible children pay up to $30 per script, or just $7.30 with a discount card.

Quoted by Minister Butler:

“Having Voxzogo on PBS can be life-changing for children born with dwarfism and their families.

“This is the first time an effective treatment is available and affordable for all families with eligible children affected by this genetic disease.

“I would especially like to commend the Murdoch Institute for Children and Professor Ravi Savarayan for leading the biomarine development program for Bokzogo, which has the largest clinical trial facility in the world.”



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