'Positive' progress funding breakthrough drug for Longford patients with rare disease
Twelve people are battling the rare disease Friedreich's ataxia in county Longford and the breakthrough treatment Skyclarys was finally approved on August 25 by the HSE Senior Management Team.
A Longford TD has said he is pleased by 'positive progression' on getting the reimbursement process set up so Longford Friedreich's ataxia (FA) patients can access the groundbreaking drug.
The breakthrough treatment Skyclarys was finally approved on August 25 by the HSE Senior Management Team after a lengthy campaign by sufferers and their families.
A total of 12 people in county Longford people are battling Friedreich's ataxia, a rare, inherited genetic disorder that causes progressive damage to the nervous system, heart, and muscles.
There are approximately 200 people living with FA across all of Ireland with approximately one in every 23,000 people in Ireland diagnosed with the disease.
County Longford is one of the counties with the highest prevalence of Friedreich's ataxia per head of population in Ireland, outside of Dublin.
Longford residents, such as Kiara Lynch from Granard, campaigned publicly to secure life-changing treatments.
Drumlish mother Theresa Kane also campaigned publicly for the HSE to fund the life-changing FA drug so her two children, Emily (14) and Ciarán (16), who were diagnosed with the rare condition, could access the only FDA-approved prescription medicine for the treatment of FA in adults and adolescents aged 16 years and older.
Deputy Micheál Carrigy said “Skyclarys was approved by the HSE for reimbursement last month.”
“Since then, work has begun on getting the reimbursement process set up as soon as possible.”
“It’s understood that the HSE aim to have the reimbursement process in place from October 1, 2026 and Biogen, the product supplier, has confirmed that it expects to have commercial stock available in Ireland from late September.”
Deputy Carrigy said reimbursement will be enabled in line with the approved European Medicines Agency market authorisation.
The local Fine Gael TD said reimbursement will be subject to prescription under the supervision of a specialist with experience of treating patients with Friedreich’s ataxia, and the prescription will be processed via the HSE High Tech Hub.
Deputy Carrigy said the HSE is working through the processes required to enable relevant specialists to register with the High Tech Hub so that their prescriptions can be processed via the Hub.
“I have been engaging with constituents since January regarding Friedreich’s Ataxia and Skyclarys. I have kept up to date on the processes that were taking place and spoken with Biogen numerous times prior to the approval for reimbursement.”
“I’m delighted to see this positive progression, however, I urge that the process is conducted efficiently so that the medication is reimbursed on time as promised,” Deputy Carrigy concluded.