Families in Northern Ireland Win Right to Spinraza Treatment

Last week we reported that three families in Northern Ireland were fighting for their children to receive the new SMA drug Spinraza (nusinersen).

Find out how Spinraza works in treating SMA patients. 

Because the drug is not yet fully licensed in Ireland, the families were told that the government was having problems finding the highly skilled clinicians needed to administer it. The problem was a matter of availability; Biogen, the drug company behind Spinraza, is providing the drug for free under its Extended Access Program (EAP) until it’s licensed.

But the BBC has an update: the families have since learned that their children will now be treated with the drug and that facilities have been made available for Spinraza to be administered via lumbar puncture.

Spinraza was approved by the U.S. Food and Drug Administration (FDA) in December 2016 and has shown remarkable promise in clinical trials for children with spinal muscular atrophy (SMA). It’s helped them to reach developmental milestones that would normally have evaded them.

SMA families in Canada are eagerly awaiting approval of Spinraza but worry about the cost. 

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