Subject to successful sign off, nusinersen is expected to be routinely available in Scotland for those with SMA Types 1, 2 and 3 from April 2019

12 February 2019

Nusinersen has been routinely available to children with SMA Type 1 in Scotland since May 2018. This will continue. The Scottish Medicines Consortium (SMC) has now granted nusinersen ultra-orphan designation, allowing the treatment to be reviewed for those with SMA Types 2 and 3 through its new ultra-orphan appraisal process with the prospect of it being made available for at least three years while further outcome data is gathered.

Pharmaceutical company Biogen will now be working with the SMC and all stakeholders in Scotland to finalise the data collection required to aid the SMC’s future assessment of the value of nusinersen.

You can read Biogen’s community statement here.

Dr Sheonad MacFarlane’s daughter, Eilidh, nine, has SMA Type 2. Sheonad, from Glasgow, also chairs Muscular Dystrophy UK’s Scottish Council. She said:

“Naturally, the Scottish Medicines Consortium’s original decision for restricted use was a huge disappointment. But we vowed to continue fighting, and the decision to make it available for people with all types of SMA now feels like we have been given a second chance. Having access to Spinraza means so much to us and to other families, and we are overjoyed that our dreams have finally been realised

“As a parent, it’s heart-breaking to see your child gradually lose their physical abilities. Nothing can prepare you for the devastating emotional impact this has on you, particularly when there is a treatment out there that is agonisingly out of reach. But now we can look to the future.”

Amy Cameron’s three-year-old son, Zac, has SMA Type 1 and has access to Spinraza on the NHS in Scotland. Amy, from Alloa, Clackmannanshire, said:

“Our whole life was thrown into a whirlwind the day we heard those three words – spinal muscular atrophy. But Spinraza has bought us valuable time with our little boy and given us hope for the future. Zac has achieved things we never thought possible and continues to amaze us every day.

“I’m so pleased the Scottish Medicines Consortium has done the right thing and approved Spinraza for people with other types of SMA. It’s made such a difference to our lives, and other families deserve that same chance.”

Doug Henderson, SMA UK’s Managing Director commented: “This is great news for the SMA community in Scotland giving wider access to this potentially life changing treatment. We now want similar access across the rest of the UK. NICE is key to this. We want NICE to recommend nusinersen for all with SMA Type 1, 2 or 3 as a matter of urgency. We also want to see a future alignment of the NICE appraisal system with this new Scottish ‘ultra -orphan pathway’ which is so much better suited to medicines for rare conditions.”

Muscular Dystrophy UK’s Chief Executive, Catherine Woodhead, says: “Today’s announcement is fantastic news for families in Scotland and gives them hope for the future. We know that Spinraza slows down the progression of spinal muscular atrophy, a devastating and sometimes-fatal condition, and this decision has the power to change the lives of up to 100  children and adults and their loved ones.

We now want to see a similar assessment route introduced in the rest of the UK, which is lagging behind Scotland and other countries in approving the treatment. Spinraza is widely available in Europe, but if a child is diagnosed with even the most severe form of the condition they cannot access this treatment in England, Wales or Northern Ireland.”