AINEWS
Skyclarys, a €280,000 (~$300,000) drug for Friedreich's ataxia, sits outside Ireland's reimbursement, and Fine Gael met Biogen Aug 17 to push compassionate access (a free or reduced cost supply from Biogen before public funding) before the HSE
About 200 people in Ireland live with Friedreich's ataxia, a rare inherited condition in which a faulty gene progressively damages the nerve pathways that control movement, balance, speech, and heart function, and which shortens life expectancy. The only approved disease-modifying drug, Skyclarys (omaveloxolone), is priced at about €280,000 per patient per year, or roughly $300,000. Ireland's Health Service Executive (HSE) has so far declined to fund it, after the National Centre for Pharmacoeconomics (NCPE) recommended against reimbursement in December 2025.
On Monday, August 17, 2026, Fine Gael TDs (members of the Irish parliament, the Dáil) and Senators met Biogen, maker of Skyclarys, in a delegation that included TDs Carrigy, Callaghan, Kyne, Maxwell, McCarthy, and O'Shea, along with Senator O'Donovan. Their ask was not a new law or a budget vote. It was a compassionate access programme, a manufacturer-led arrangement in which a drug company provides its medicine free or at reduced cost before public reimbursement is in place. The near-term trigger was August 25, when the HSE's senior management team can revisit the December NCPE recommendation at a structured review point.
Compassionate access is a separate, manufacturer-led pathway. The HSE funds medicines only after the NCPE runs a health-technology assessment and a price-versus-benefit calculation closes; a "no" can be reopened at a senior management meeting, but only within that system. Compassionate access sits outside the reimbursement process, which is why the political delegation was asking Biogen directly rather than asking the HSE to override its own process.
Skyclarys is approved, but its evidence base is contested. The U.S. Food and Drug Administration cleared the drug in February 2023 and the European Commission followed in February 2024, making it the first disease-modifying therapy for Friedreich's ataxia. Patients and advocates say it slows disease progression by up to 55 per cent; the European Medicines Agency's assessment, the NCPE, and the HSE Drugs Group have all flagged limitations and uncertainties in the underlying clinical efficacy data, and the HSE's Drugs Group upheld the NCPE recommendation on those grounds.
The cost-benefit calculation did not close at €280,000 per patient per year (~$300,000), even though the drug is approved, the disease is recognized, and roughly 200 Irish patients are eligible. Belgium, Sweden, and the Netherlands have publicly flagged similar real challenges with Skyclarys's efficacy and impact, a cross-border pattern that places the Irish refusal in a wider context rather than as an outlier.
The political pressure crosses party lines. Sinn Féin leader Mary Lou McDonald has written to Taoiseach (Irish prime minister) Micheál Martin, and a majority of the Fianna Fáil parliamentary party has called on the HSE to reconsider, according to The Journal.
The August 25 HSE senior management meeting can move three things: uphold the December 2025 recommendation, ask Biogen to come back with a lower price or a managed-access arrangement, or send the file back for further review. Without a price change, a managed-access deal, or a new public funding mechanism, it cannot on its own fund a €280,000-a-year drug for 200 patients. The compassionate access ask is directed at Biogen, not the HSE, because Biogen sets the price, and the August 25 review can only adjust Ireland's response to it.
The HSE review on August 25 will set the next public position; until then, Biogen's response to the compassionate access ask is the decision that does not wait for the system.