Pricing agreement on life-changing treatment for Spinal Muscular Atrophy welcomed by SMA Ireland
HSE to reimburse for single-dose Zolgensma gene therapy to treat babies with SMA
SMA Ireland, the patient representative group for people affected by Spinal Muscular Atrophy (SMA) has today (08.10.21) welcomed the announcement of a pricing agreement for Zolgensma, a gene therapy for the treatment of SMA. Negotiated by Belgium, Ireland and the Netherlands through the BeNeLuxA Initiative, the agreement means that the HSE will now provide reimbursement for this single-dose treatment for new-born babies with SMA type I, or pre-symptomatic children with up to three copies of the SMN2 gene.
Commenting, Director of SMA Ireland, Jonathan O’Grady said: “This is a hugely significant day for Irish babies with SMA as well as their families, carers and friends. I would like to congratulate the teams involved in the negotiation process from the HSE, the Department of Health and the National Centre for Pharmacoeconomics for working in harmony with European partners to get this deal across the line.
“This is an excellent example of how collaborative negotiation with pharma companies can work to deliver quick, affordable, and innovative medical solutions. Credit must also be attributed to Dr Declan O'Rourke and the Children’s Health Ireland team at Temple Street Children’s University Hospital who have worked incredibly hard behind-the-scenes securing access to this treatment.”
Zolgensma was developed by Novartis Gene Therapies and approved by the European Medicines Agency in March 2020.
Mr O’Grady added: “Today’s 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.
“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. Detection at the earliest point is absolutely critical here to achieve the best treatment outcomes for babies with SMA.”
SMA is a rare, progressive, neuromuscular disease which leads to immobility and results in a short life expectancy for many children diagnosed with the condition. Approximately one in 8,000 people are diagnosed with SMA depending on the country. SMA is caused by a homozygous deletion of the SMN1 gene.
The disease involves the loss of nerve cells called motor neurons that control muscles. Once lost, motor neurons cannot be regenerated and up to 60 per cent of children born with SMA will never be able to sit up independently and do not live beyond two years of age. Despite being a rare disease, left untreated, SMA is the leading genetic cause of death in infants and toddlers.
Mr O’Grady concluded: “We encourage families with new-born babies missing milestones to consider contacting SMA Ireland. Our website contains further information, and our representatives are on hand to offer guidance and assistance. Over the years we have found knowledge sharing and conversation to be an exceptionally important tool as part of advocating for access to new treatments and we remain committed to supporting the lives of those affected by SMA, through awareness raising and securing access to further treatments until a cure is found.”
For further information on spinal muscular atrophy in Ireland, visit: www.smaireland.com.