The Scottish Medicines Consortium (SMC) has recommended the routine funding of Spinraza (nusinersen) for the treatment of symptomatic type 1 5q spinal muscular atrophy (SMA) (infantile onset). The acceptance comes after the SMC took just four months to review the evidence and make their decision.

SMA is a debilitating and life-threatening rare disease – it is the leading genetic cause of death in infants, with type 1 children rarely living to see their second birthday. The disease takes away a person’s ability to walk, eat and ultimately, breathe.

Following the SMC recommendation, Biogen is in active discussions with the national body to resubmit nusinersen for later onset SMA (types 2 and 3) and urges the initiation of a reformed appraisal process for ultra-orphan medicines as soon as possible to allow a greater degree of flexibility for the assessment of such treatment options. Applications for patients with later onset SMA to receive nusinersen are being made on a case-by-case basis by treating hospitals in Scotland.

Terry O’Regan Managing Director and Vice President of Biogen UK and Ireland said, “Our job is not done, and we continue to actively collaborate with patients, clinicians and government bodies – both in Scotland and also across the rest of the UK – to find a way to make this important treatment available to all who may benefit.”

In clinical trials, nusinersen was shown to significantly improve the disease across types 1, 2 and 3 SMA, with motor function improvements from the start of treatment such as independent sitting, standing and walking, as well as improvements in breathing and survival among those most severely affected.

Due to the urgent need, Biogen opened one of the largest global expanded access programmes in rare disease for children with infantile onset SMA (consistent with type 1) in Autumn 2016, even before marketing authorisation was granted. To date, Biogen has provided all eligible children in the UK with nusinersen free of charge via this programme.

Assessments of nusinersen in England and Wales remain in progress, with decisions expected later in 2018.

Terry O’Regan Managing Director and Vice President of Biogen UK and Ireland said, “Our job is not done, and we continue to actively collaborate with patients, clinicians and government bodies – both in Scotland and also across the rest of the UK – to find a way to make this important treatment available to all who may benefit.”