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Spinraza campaigners 'finally have hope' as HSE approves access to drug for rare disease

The drug has been approved for children with SMA Type I, II or III on “an exceptional and individualised basis”.

Sam and Fiona Bailey
Sam and Fiona Bailey
Image: Órla Ryan

THE HSE HAS approved access to the drug Spinraza in the treatment of children with spinal muscular atrophy (SMA) after a lengthy campaign by families and patients.   

SMA is a rare, debilitating and life-threatening disease that affects nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. 

Where it develops in babies and toddlers it can significantly reduce life expectancy. While it is not a cure, trials have shown that Spinraza, also known as nusinersen, can slow the effects of SMA in some cases.

Minister for Health Simon Harris announced this morning that the HSE would fund the drug for children only. 

“This is the first ever treatment for children with SMA. I know how hard the long journey to get here has been for families but very glad children in Ireland will now have access,” Harris tweeted. 

In a statement today, the HSE said it had approved access for children with SMA Type I, II or III on “an exceptional and individualised basis”.

“The recommendation is to approve access for children under 18 years with genetically confirmed SMA Type I, II or III, in accordance with the controlled access criteria recommended by the Rare Diseases Technology Review Committee.”

To date, more than 7,500 patients worldwide have been treated with nusinersen, from infants to adults.

The most common form of the disease is 5q SMA, which has four different types: 1, 2, 3 and 4– based on age of onset and the highest physical milestone achieved. Type 1 is the most severe form of SMA and accounts for between 50–70% of cases of childhood onset SMA. About 95% of children with Type 1 who are untreated die before the age of two. 

Fiona Bailey, whose son Sam (9) has SMA, said they are all “absolutely over the moon” at today’s news. 

“It’s an amazing day for us all, it’s a day we’ve been working towards for a long time.

“The happiest day we’ve had in a long time, we finally have some hope for Sam’s future. Students all over Sam’s school were crying this morning after hearing the news,” Fiona told TheJournal.ie. 

Sam is stronger than anyone I know. In his heart and in his soul he is a fighter. Now that Spinraza has is a reality for us the future looks brighter. 

Concern 

The HSE had previously said it would not reimburse for the drug at its current price, stating it would cost more than €20 million over a five-year period.

The drug was approved by the NHS back in May after an agreement with its makers, Biogaen, to fund treatment for a limited time to allow more data to be collected on its effectiveness. 

In today’s statement, the HSE said it will work closely with the Department of Health in relation to the funding implications of today’s decision; particularly in relation to funding for future years.

SMA Ireland had previously voiced its concern that the HSE would apply an age-based cut off to treatment to save costs.

“We genuinely hope that reason and common sense has held sway and that treatment will be available for all people with SMA,” the group tweeted earlier. 

With reporting from Órla Ryan 

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Adam Daly

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