The HSE’s Senior Management Team (SMT) has approved the reimbursement of the drug Skyclarys for Friedreich’s Ataxia patients a fortnight after the HSE Drugs Group recommended against its reimbursement.
It follows a long-running and emotional campaign led by some of those with the rare progressive neurodegenerative disorder, which affects around 200 people in Ireland.
A recommendation from the HSE Drugs Group, issued earlier this month, not to reimburse Skyclarys, the first approved treatment for Friedreich’s ataxia, sparked backlash and demonstrations.
Campaigners pointed out how Cyprus, Italy, Latvia, Spain, Portugal, Luxembourg, France and Germany in the EU all fund medication to treat the disease – however, the Irish State did not deem the medication value for money.
Pharmaceutical company Biogen and the HSE have had over two years to reach a decision on the reimbursement of the drug. The HSE said today that the decision followed a “substantially improved financial offer” from Biogen.
A protest in Dublin City Centre on Saturday was attended by more than one thousand people, while last week saw a large silent walk through Mallow in Cork in support of Buttevant father Craig Coady, his son Rory, and the more than 200 families waiting for access to the drug.
The rare disease causes progressive damage to the nervous system. In 2024, the drug omaveloxolone, branded as Skyclarys, was the first licensed drug approved in Europe for treatment of the disease.
Advocates and patients say that while it is not a cure, it has the ability to slow the progression of Friederich’s ataxia by up to 55 per cent.
Today, August 25, was the deadline for a decision on whether the State would reimburse the drug.
Ataxia Foundation Ireland said it was a landmark day for the Friedreich’s Atazxia community in Ireland.
“Today’s decision offers real hope to people living with Friedreich’s ataxia and the families who support them every day,” it said.
“We recognise the constructive engagement that has taken place to secure this outcome, including the improved commercial offer made by Biogen. We also welcome the HSE’s recognition of the significant unmet need faced by people living with Friedreich’s ataxia and the absence of other medicines to treat the condition.”
“This follows months of tireless campaigning by people living with Friedreich’s ataxia, their families, clinicians, advocates, and supporters. Every person who shared their story, contacted a public representative, joined the demonstration in Dublin or supported the campaign helped ensure that this community was heard.”
Emily Felix, a 28-year-old with the rare condition, who has become one of the most prominent campaigners alongside Mr Coady, today said the news was “amazing.”
On the eve of the decision, Ms Felix said that patients had become “angry” and “exhausted” and that they would “not stop fighting until [their] voices are heard.”
“It’s been a very long battle. Over two years waiting – this shouldn’t have ever had to be this hard, this fight that we’ve put in,” said Ms Felix, who is from Kilkenny.
“But I’m incredibly proud of our whole community, our family and friends, and everyone who has fought so hard with us to get to this stage where we finally have the answer we need, which is the approval for everyone in our community,” said the trainee solicitor.
“Everyone has a quality of life, and they deserve to maintain it and preserve their abilities. This should have been done a long time ago, but we are delighted and over the moon with this decision today.”
Independent Ireland today welcomed the decision having previously written to TD Padraig Rice, Chair of the Health Committee, requesting that the committee be immediately reconvened in light of the HSE recommendation not to approve the drug for patients in Ireland.
Cork TD Ken O’Flynn said that the families had “earned” the outcome.
“This is a victory that belongs to the families. To the roughly 200 people across Ireland living with this progressive condition, and to the campaigners who refused to be ignored,” said Deputy O’Flynn.
“I want to pay particular tribute to Niamh Ní Hoireabhard, Emily Felix and Aoife Gavan, who led Sunday’s demonstration in Dublin, and to every person who stood up, went public and shared their story. It is never easy to put your own life and your own diagnosis in front of the country. But it worked. Your courage changed minds and, ultimately, changed a decision.
“I also want to acknowledge Craig Coady, who played a vital role in all of this. His work and his determination were central to getting us over the line, and this outcome would not have happened without him.
“Skyclarys is not a cure. But patients and advocates tell us it can slow the progression of this disease by up to 55%. For a family watching a loved one’s health decline, that is not a statistic it is time. It is hope,” he added.
“To the families: today is your day. You earned this,” added O’Flynn, who said his party would “keep fighting for fair access to medicines, for rare disease patients, and for a health service that puts people first.”
Peadar Tóibín, leader of Aontú, said the government “need to get their priorities right in future.”
In a statement, the Minister for Health said it was an important day for those with Friedreich’s Ataxia and their families, and an “important day in a world where their disease brings far too much uncertainty.”
“Though I know the statutory process through which these decisions are made is complex, it is important that politics and politicians allow the space for science to be the decision maker,” said Minister Jenifer Caroll MacNeill.
“At a time when there is too much uncertainty in the world, Ireland will continue to stick to science and to the rule of law. Simply put, these processes must be independent and led by experts.
“I am grateful to our Health Service Executive (HSE) scientists and to our commercial team involved in the negotiations for their continued and active engagement to reach today’s decision and I thank the manufacturer for their intensive engagement, especially in recent days.
“I am grateful to the patients who have so carefully articulated their lived experiences. I am also grateful to the many clinicians who provided the HSE’s Senior Management Team with their expertise and advice to make their decision today.”
The Minister said that the HSE would now engage with the company to put in place the process by which patients can gain access to the drug.