Friedreich’s ataxia: What is it, and how can Skyclarys help patients?

So what is Friedreich's ataxia and how does Skyclarys help those who have the condition?

What is Friedreich's ataxia?

Friedreich's ataxia is a disease that damages the spinal cord, peripheral nerves and the cerebellum, a part of the brain.

It can also lead to heart problems and gradually worsens over time.

Those who have the disease can experience unsteady, awkward movements and a loss of feeling due to nerve injury, which develops as the condition progresses. They may also have other health problems, including diabetes and heart disease, along with other nervous system symptoms.

How many people have the disease in Ireland? 

It is believed that there are around 200 people with the condition across the country.

A long campaign fronted by families, including Craig Coady, who has already lost one son to the disease, has focused political and media attention on Skyclarys in recent months.

What is Skyclarys? 

It is a medicine used to treat patients aged 16 and older with Friedreich's ataxia, according to the European Medicines Agency.

While it is not a cure for the condition, it helps slow the progression of the disease. This includes slowing its impact on the vocal cords and voice.

Skyclarys was first approved by the European Medicines Agency in 2024. The evaluation for its reimbursement in Ireland began more than two years ago.

How does Skyclarys help patients?

Skyclarys is not a cure for Friedreich’s ataxia, but it can slow the physical deterioration associated with the condition.

The treatment can help slow the progression of symptoms, including the impact of the disease on patients’ voices and vocal cords.

Maureen Sweeney, head of operations at Ataxia Foundation Ireland, described the decision to fund the drug as “absolutely life-changing” for some patients.

She said the drug's impact on speech was particularly significant because it could help patients continue to communicate for longer.

Why was funding initially rejected?

The HSE Drugs Group had earlier advised against reimbursement of Skyclarys, citing concerns about the cost of the treatment and the limited clinical evidence of its effectiveness.

The HSE said its senior management team took account of these concerns when considering the revised commercial offer from Biogen.

However, it also considered the “substantial unmet need” among patients with Friedreich’s ataxia and the fact that there are no other medicines available to treat the condition.

Who can receive Skyclarys?

Skyclarys is licensed for use in patients aged 16 and over with Friedreich’s ataxia.

According to Ataxia Foundation Ireland, the treatment is being licensed in Ireland under the European Medicines Agency approval, meaning it will be available to eligible patients aged over 16.

Why is the decision significant for families?

Families affected by Friedreich’s ataxia have campaigned for access to Skyclarys for months.

Craig Coady, whose son Paudie has the condition and whose other son, 13-year-old Rory, died from the disease last September, said the HSE decision was “like a ton weight had been lifted”.

“I feel exhausted now. Hopefully it won't be too long to get the drugs into our hands,” he said.

What happens now? 

The HSE has approved funding for Skyclarys following a substantially improved financial offer from Biogen.

The HSE and Biogen are now working to finalise arrangements for the rollout of the drug to patients and the administrative systems required.

Health minister Jennifer Carroll MacNeill has asked that this work begin immediately, saying she expects the HSE and Biogen to work together to ensure patients can get swift access to the treatment.

“The HSE will progress these engagements quickly, and understands that Biogen is also committed to finalising details at an early stage,” the HSE said.