THE approval for a gene therapy drug for young children with Spinal Muscular Atrophy means that children born with the disease will now have access to the life-saving drug.
That is according to the director of SMA Ireland, Jonathan O’Grady, following approval being granted for Zolgensma to be reimbursed.
One child in Cork is suited to the drug – Kate Mynard from Dunmanway. Her case, along with that of Kerry child Theo Whelan, was brought up in the Dail by Fianna Fail TD Christopher O’Sullivan.
The single-dose treatment is for newborn babies with SMA type I, or pre-symptomatic children with up to three copies of the SMN2 gene. SMA is a rare genetic muscular condition leading to loss of movement.
“This is a hugely significant day for Irish babies with SMA as well as their families, carers and friends," Mr O’Grady said.
He added that the “announcement means that the two or three children born each year in Ireland with the most severe form of the disease will now have access to this life changing medicine.”
“However, to ensure that Zolgensma will have maximum effect for those who meet the criteria, it is vital that treatment is administered as early as possible," he said. "We are therefore calling for SMA to be added to the national list of diseases assessed as part of the neonatal screening process.
"Genetic screening for common and rare disorders, including SMA, is now standard practice in other European countries and in Australia, Canada, and the United States. The list of diseases screened in Ireland is currently under review by Professor Niall O'Higgins and the National Screening Committee.
“I’m delighted for the families," Deputy O’Sullivan said. "Well done to them for highlighting this issue. This treatment will give baby Kate and baby Theo a really improved outlook and quality of life.
He added: “I’m so glad we got a good outcome for these two children. I want to thank both the Taoiseach and Minister Donnelly for listening to the pleas of both myself and the families.”
He said it is the first time Ireland has agreed to fund a drug for rare diseases in the BeNeLuxA Initiative.
The BeNeLuxA Initiative is a partnership approach to pharmaceutical policy with Belgium, the Netherlands, Luxembourg and Austria that aims to secure sustainable access to high quality and affordable medicines in the participating countries.