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Friedreich’s ataxia (FA) drug Skyclarys approved by TGA

The first ever treatment for Australians with a rare neurodegenerative disease has been approved by the watchdog.

The treatment offers hope for Tom Koenig, pictured with parents Rohan and Amanda and brother Lochie. Picture: Richard Walker
The treatment offers hope for Tom Koenig, pictured with parents Rohan and Amanda and brother Lochie. Picture: Richard Walker

The first ever treatment for Australians with a rare neurodegenerative disease could help slow the progression of its damage to the nervous system.

The Therapeutic Goods Administration approved the use of Skyclarys (omaveloxolone) for Australians aged 16 and above with Friedreich’s ataxia this month, which affects one in 30,000 people.

An inherited disease that targets the central nervous system, FA causes walking difficulties, fatigue, slow or slurred speech, and loss of reflexes and sensation in the legs, arms, and body.

Scoliosis and heart disease symptoms can also develop over time.

Symptoms usually begin between the ages of five and 25, but can appear in younger children or adults in their 30s or 40s.

Many patients lose the ability to walk within eight to 10 years of diagnosis.

Royal Brisbane and Women’s Hospital neurologist and FA expert Joel Corbett said Skyclarys slowed the progression of the disease and targeted damage to the nervous system.

“With Friedreich’s ataxia, the genetic abnormality causes accumulation of damaging products in the mitochondria, which are the energy-producing cells in the body,” he said.

“The drug is helping to increase the defence mechanisms against this oxidative stress in the mitochondria, and would reduce the amount of damage to the nervous system.”

Dr Corbett said the drug, though not curative, would offer a better quality of life for patients.

“It will provide people living with FA a sense of hope of a medication that might help to give them a little bit more stability in their lives,” he said.

“With the progression of the disease each year, things get a little bit harder for them and their independence becomes gradually worse … so it isn’t going to prevent people from serious disability but … it allows them to have a better quality of life for a longer period of time.”

A Royal Brisbane and Women’s Hospital neurologist says the drug can offer better quality of life.
A Royal Brisbane and Women’s Hospital neurologist says the drug can offer better quality of life.

Dr Corbett said current therapies were focused on offering support and managing symptoms, like walking aids or wheelchairs, and dietary modifications to help patients struggling to swallow.

“There is not any other specific medication treatments except for people who have heart disease associated with the condition,” he said.

The newly approved drug offered 16-year-old Brisbane boy Tom Koenig more time to have normalcy in his life.

Father Rohan Koenig said the family had spent years trying to find answers for Tom, who started off with co-ordination difficulties when he was four.

He was diagnosed at 10 years old.

“He loses a few things he loves each year … he was playing Auskick but eventually couldn’t run up and down the field. Same thing with his cricket, he played that up until he was in grade seven,” Mr Koenig said.

The Kenmore Hills father said the announcement of the drug’s approval in Australia was a good step for his son, who enjoyed studying biology and playing wheelchair basketball.

“If it can buy us time, it is good. Because it is progressive and you lose things each year, it just makes it hard for Tom. It is the first treatment ever, which is perfect and it gives us time to work towards what will eventually be a cure,” Mr Koenig said.

“Tom is at that lucky point with these drugs that are coming through, it can help him have a more normal life going forward.”

If Australians were experiencing issues with balance or co-ordination, Dr Corbett said it would be appropriate to seek advice from a GP.

Originally published as Friedreich’s ataxia (FA) drug Skyclarys approved by TGA

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Original URL: https://www.heraldsun.com.au/health/conditions/friedreichs-ataxia-fa-drug-skyclarys-approved-by-tga/news-story/af1fe910472c68ba65fc0b14cf70469e