The HSE Drugs Group has decided to recommend that the drug Skyclarys, for Friedreich's Ataxia, should not be covered by the HSE.
The group had initially recommended that Skyclarys not be covered, however, it met to consider a report from the Rare Diseases Technology Review Group which supports the introduction of Skyclarys.
The recommendation will now go to a meeting of the HSE senior management team on 25 August for a final decision.
Around 200 people are living with the rare neurological disease here and have been campaigning for the drug to be made available by the HSE. It is the first treatment for Friedreich's Ataxia.
Campaigners for people with the condition said they were heartbroken with the news from the HSE Drugs Group and were absolutely devastated.
They have been seeking a meeting with the Minister for Health Jennifer Carroll MacNeill and have been lobbying politicians. The campaigners added that they will not give up fighting.
In a statement, the HSE said that in making their recommendation, the HSE Drugs Group examined submissions and data from Friedreich's Ataxia patients, their doctors and advocacy groups.
It said the group discussed the profound and devastating impact that Friedreich’s Ataxia has on quality of life and life expectancy.
The HSE added that among the issues when considering any drug, it looks at the cost of the drug, and the impact providing that drug would have on the overall resources available to the HSE, and the finite budget available for new medicines.
In making its decision on whether a drug should be available on the public system, the HSE Drugs Group considers reviews by the National Centre for Pharmaeconomics, patient group submissions and the outcome of commercial negotiations with relevant drug companies.
Last December, the National Centre for Pharmaeconomics recommended against the HSE providing the drug as it was not seen as cost-effective.
At the current price, it would cost around €280,000 per patient per year, with a five-year budget impact of around €130m.
The drug is made by Biogen which has said it continues to engage with the HSE.
Skyclarys has been approved by the European Medicines Agency and the FDA in the US.
Earlier this month, the High Court gave Emily Felix, one woman with Friedreich's Ataxia, permission to challenge what she said is the HSE delay on granting her access to the drug.
