Hope for children with rare disease as health chiefs recommend approval for lifesaving drug

Duchenne Muscular dystrophy (DMD) is a severe progressive genetic disorder that affects around 100 children in Ireland

Archie Ennis is taking part in a clinical trial for gene therapy in the US, but needs Givinostat to ensure he stays on his feet and out of a wheelchair.

The HSE Drugs Group has recommended that a life-changing drug for children with a rare disease should be approved.

Fianna Fáil Senator Teresa Costello confirmed on Wednesday evening that the HSE Drugs Group had recommended that Givinostat should be provided to those with Duchenne Muscular Dystrophy.

Duchenne Muscular dystrophy (DMD) is a severe progressive genetic disorder that weakens muscles. It affects around 100 children in Ireland.

Ms Costello said that the HSE Drugs Group, which makes recommendations on the pricing and reimbursement of medicines, met earlier this week and has now made a recommendation to the HSE leadership team to approve Givinostat.

“This is a positive day for every individual and family impacted not only by Duchenne Muscular Dystrophy, but every rare disease,” she said.

“I want to thank the families for their belief and perseverance, even on the toughest of days.

“I also want to thank the Taoiseach [Micheál Martin] and [health] minister [Jennifer] Carroll MacNeill for their constant engagement throughout this process, not only with me, but also with families impacted by the disease.

“Finally, I want to thank my colleague, Deputy Pádraig O’Sullivan, who has been a champion for rare diseases, for his support."

Ms Costello concluded that she’s looking forward to the next step in the process and ensuring that the drug gets to children who need it.

This includes 10-year-old Tallaght child Archie Ennis, whose mother Una told the Irish Examiner earlier this week that she felt her child’s life was “in the hands of the HSE”.

Archie is taking part in a clinical trial for gene therapy in the US, but needs Givinostat to ensure he stays on his feet and out of a wheelchair.

Ms Ennis and her husband Kenneth fundraised money to get the €3.2m gene therapy for Archie. The Ennis family moved to the US in January and stayed there for four months as Archie took part in a clinical trial. They now must return once a month, for three months.

“We wish we could have had it in our own country, to be at home,” Ms Ennis said.