Scotland leads the way in approval of first licensed treatment for life-threatening genetic disease affecting children

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Spinraza

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, marking the first ever treatment option available to those with the most severe form of the disease.

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.

“Biogen is extremely proud to bring nusinersen to patients in Scotland. SMA is a devastating condition in many cases, and this treatment is making a momentous difference in the lives of those who previously had no other options,” comments Terry O’Regan, managing director and vice president of Biogen UK and Ireland. “We are very pleased that the SMC has acted quickly, and that SMA type 1 patients will now have the reassurance that a treatment will be available to them moving forward.”

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. Reforms were proposed to the Scottish Government in 2016 following a review of the system and the Cabinet Secretary for Health and Sport confirmed that the Scottish Government would take forward the recommendations. Biogen hopes this new, more appropriate process will lead to a positive outcome for all who may benefit from nusinersen. In the meantime, 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 continues, “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. Our commitment to SMA patients remains unfaltering, and we stand ready to play our part.”

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, 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, and Biogen is working closely with the relevant authorities to expedite the timelines where possible to bring the treatment to patients without delay. Elsewhere, patients across disease types 1, 2 and 3 have access to nusinersen in Italy, France, Germany, Norway, Austria, Switzerland, Spain, Israel, Slovenia and Luxembourg, while In Denmark, infantile onset SMA patients have access.

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