Cuts to cost of dwarfism drug to save families millions

·2-min read

At only three months old, Oscar Rose was rushed to hospital for what ended up being emergency spinal therapy.

The now 10-year-old was born with achondroplasia, the most common form of dwarfism.

His mother Hanna was forced to resuscitate him 20 times over the years due to breathing complications, but a new drug has helped cut out serious hospital visits.

Voxzogo will now be listed on the Pharmaceutical Benefits Scheme, reducing the cost of the drug from $330,000 a year to $30 a script, or just over $7 for concession card holders.

The treatment plan would usually last until the mid to late-teens, meaning millions of dollars would have otherwise been needed to continue on the drug.

More than 250 children worldwide are participating in trials for the drug's active ingredient, vosoritide, which is proving to be safe and effective.

Ms Rose said the treatment had helped the family remove aids around the house and given her son more independence.

It's enabled him to grow at the same rate as a normal pre-teen.

"This is life-saving and life-changing," she said. 

It's also made life easier for Naoko Abe, who had to fly to Japan every fortnight to get treatment for her son Ari.

While the cost of the trial was covered, the family spent thousands on flights.

"The bank account balance has gone low, but you will do it," she said, welcoming the announcement alongside her son in Canberra on Wednesday.

The 12-year-old has grown four centimetres since December, meaning he can also access more things around the house, increasing his independence.

Professor Ravi Savarirayan said dwarfism was more than a condition affecting height, it also put pressure on spinal cords and led to other life-long complications.

He said the drug was a game-changer and enabled those born with achondroplasia to live a normal life.

They are almost 50 times more likely to die before turning five than other children.

"For many years, I was left in embarrassed silence when patients would ask me: 'What can you do about my child's problem?'," the specialist said, noting the gene had been found 30 years ago.

"We are trying to increase lifelong health for these children so that they can have increased independence, better functionality, decreased complications and hopefully put me out of a job."

Achondroplasia affects around one in 20,000 babies in Australia.  

Voxzogo has been approved in more than 37 countries.