Campaigners gathered in Dublin city centre over the weekend in what they are calling a final push to get the HSE to approve reimbursement for Skyclarys, a drug used to treat Friedreich’s Ataxia.
Friedreich’s Ataxia is a rare, progressive neurological condition that affects movement and coordination. It typically begins in childhood or early adulthood, gradually robbing people of their ability to walk, and in many cases affecting the heart. There is no cure, which makes access to disease-modifying treatments all the more critical for those living with the condition.
Skyclarys, known generically as omaveloxolone, is the first approved drug specifically targeting Friedreich’s Ataxia. It received approval from the European Medicines Agency, raising hopes among patients and their families that it would become accessible across EU member states, including Ireland. But getting a drug approved and getting it funded through the public health system are two very different battles.
The HSE has yet to approve reimbursement for Skyclarys, meaning patients in Ireland cannot access it through the medical card or drug payment scheme. For most families, that puts the treatment completely out of reach financially.
That frustration boiled over on the streets of Dublin as campaigners, many of them patients and family members, turned out to demand action. Demonstrators made clear they are tired of waiting and want a decision from the HSE without further delay.
Campaigners have argued that every month without a decision is a month of progression for patients who cannot afford to wait. Friedreich’s Ataxia does not pause while committees deliberate, and for younger patients especially, delays can mean permanent losses in function that might have been slowed with earlier treatment.
Ireland has a well-documented history of lengthy delays in approving reimbursement for rare disease treatments. Families dealing with conditions like Friedreich’s Ataxia often find themselves in an exhausting cycle of hope and disappointment, watching drugs get regulatory approval only to sit in a reimbursement queue for months or years.
The campaign around Skyclarys has been building for some time, with patient advocacy groups pushing the issue through political channels as well as public demonstrations. The Dublin rally was framed as a final push, suggesting campaigners believe a decision may be close and want to keep pressure on the HSE at what they see as a critical moment.
Support has been growing in political circles too, with several TDs raising the issue in the Dail and calling on the Minister for Health to intervene and speed up the process. The broader conversation around rare disease drug access in Ireland has intensified in recent years, with families increasingly willing to go public with their stories to force the issue into the spotlight.
For those living with Friedreich’s Ataxia in Ireland, the stakes could not be higher. Skyclarys is not a cure, but it represents a genuine step forward in managing a condition that currently has very limited treatment options. Campaigners say that approving reimbursement is not just a healthcare decision, it is a statement about whether the Irish health system values the lives of people with rare diseases.
The HSE has not yet indicated when a final decision on Skyclarys reimbursement will be made.