Ireland approves funding for first Friedreich’s ataxia medicine

Ireland’s Health Service Executive has approved public reimbursement for Skyclarys, opening a route to the first medicine for people with Friedreich’s ataxia in the country. The rare, progressive neuromuscular condition damages nerves and can cause muscle weakness and loss of mobility. About 200 people in Ireland are estimated to live with it. Skyclarys is not a cure, but it can slow progression.

The decision followed intensive negotiations and a substantially improved financial offer from manufacturer Biogen. Earlier reviews had recommended against reimbursement because of cost-effectiveness concerns and questions about the strength of the clinical evidence. The HSE said the revised terms created a materially different proposal. Its senior management team still noted limited effectiveness and a high remaining cost, but weighed those concerns against the severe unmet need and the absence of any other medicine for the disease. The final negotiated price remains confidential.

The approval follows sustained campaigning by patients and families, who had faced weeks of uncertainty after the earlier negative recommendation. Work is now under way between the HSE and Biogen to complete the reimbursement systems and rollout arrangements. Ireland’s health minister has asked both sides to begin that work immediately, while advocates are pressing for treatment to reach eligible patients without further delay.