Negotiations must be stepped up for breakthrough drug for rare genetic disease – Carrigy

Urgent progress is needed between the HSE and pharmaceutical company Biogen on making the treatment for a rare neurodegenerative disease publicly accessible, a Fine Gael TD has said.

Deputy Micheál Carrigy met recently with Longford families affected by Friedreich’s Ataxia and then met with Biogen – the manufacturer of the treatment – to push the case for public access to the drug.

Deputy Carrigy outlined: “Following that meeting and contact with Biogen, its officials confirmed to me they have sent proposals to the HSE following their initial engagement in February.

“These drugs are developed to help people. I don’t believe that the cost should be an impediment in making life-changing differences for people’s lives.”

Around 200 people in Ireland live with Friedreich’s Ataxia, a rare inherited neurodegenerative disorder which causes progressive damage to the spinal cord and peripheral nerves, which leads to impaired muscle co-ordination, loss of balance and difficulties with speech, vision and hearing.

Skyclarys is the first approved treatment shown to slow the progression of the condition, with some doctors saying it can slow the rate of progression by up to 50%.

The drug was approved in Europe in February 2024 with Spain, France and Italy already reimbursing the cost through their health services.

“There needs to be action on this,” continued Deputy Carrigy. “The National Centre for Pharmacoeconomics recommended at the end of last year that Skyclarys be considered for reimbursement.

“However, the HSE application remains under consideration and a final decision has yet to be made.”

“I will be following up with both the HSE and Biogen to ensure negotiations progress without delay. The 200 people in Ireland living with the disorder can’t afford to wait, with every month and year meaning further progression of the condition.”