For families living with Friedreich’s ataxia, every single day matters. The rare neurological disease slowly robs people of their ability to walk, move, and eventually breathe on their own. Now, patients and their loved ones are putting serious pressure on the HSE to fund Skyclarys, the first approved treatment shown to slow the progression of the condition.
Friedreich’s ataxia affects roughly one in every 50,000 people. It is a genetic disease that damages the nervous system and the heart over time, typically showing up in childhood or early adulthood. There is no cure. Until recently, there was not even a treatment that could meaningfully slow it down.
Skyclarys, known generically as omaveloxolone, changed that. It became the first drug approved in Europe specifically for Friedreich’s ataxia, offering patients something they had never had before, a real chance to hold on to their physical function for longer. Clinical trials showed it could slow neurological decline in patients aged 16 and over, which for many families feels like nothing short of a lifeline.
But in Ireland, that lifeline is sitting just out of reach. The HSE has not yet agreed to reimburse the cost of the drug, meaning patients here cannot access it through the public health system. For a condition that progresses regardless of whether treatment is available or not, the wait is not just frustrating. Families say it is devastating.
Patients and advocacy groups have been vocal about the urgency of the situation. The message coming from the Friedreich’s ataxia community is blunt and heartbreaking in equal measure. They are not asking for miracles. They are asking for a chance. A chance to slow things down, to keep walking a little longer, to hold onto independence that the disease is quietly taking away.
The drug is already approved and available in other European countries. Ireland risks falling behind in giving its patients access to a treatment that has gone through the full regulatory process and has been deemed safe and effective. For rare disease communities, that kind of gap in access hits especially hard. These are already small groups of people who have spent years with very few options, and when something finally comes along, the funding delays feel like a second blow.
Skyclarys is not cheap. Treatments for rare diseases rarely are, and the cost is a significant factor in any HSE reimbursement decision. The health technology assessment process exists for good reason, and balancing drug costs against outcomes is a genuine challenge for any public health system. But families affected by Friedreich’s ataxia argue that the cost of inaction is also real, just harder to put a number on.
The disease does not pause while the paperwork is processed. Young people who could potentially benefit from slowed progression are getting older, and in some cases, moving past the window where the drug is most effective. Time is not a neutral factor here. It is the whole point.
Advocacy groups are calling on the HSE and the government to fast-track a decision and make Skyclarys available to Irish patients without further delay. They want to see the kind of urgency applied to this process that matches the urgency families are living with every day.
For parents watching their children lose physical function, or for young adults trying to hold onto their independence, the ask is not complicated. Fund the treatment. Give people a fighting chance. The science has done its part. Now they need the system to do its.