Children with the most severe form of a devastating genetic condition will have immediate access to a life-changing treatment, with a landmark decision to make it available on the NHS in Scotland announced today. However, there is disappointment for patients with other variations of the condition, as the same treatment will not be offered to them.
Spinraza is the first and only treatment for patients with the rare inherited muscle-wasting condition spinal muscular atrophy (SMA), and today (Monday 7 May), the Scottish Medicines Consortium (SMC) recommended that it should be made available on the NHS in Scotland for children with Type 1 SMA. However, it did not approve the treatment for patients with Type 2 and 3 of the condition.
Muscular Dystrophy UK has been working in partnership with other SMA charities, SMA Support UK and The SMA Trust, to push for access to Spinraza for all patients with the condition. Without it, life expectancy for those with SMA Type 1 is rarely longer than two years, but with treatment, children can learn to crawl and even walk.
The charities welcome the news that Spinraza will be available to SMA Type 1 patients, but will now be pushing for a reassessment of the treatment for all types of SMA. Muscular Dystrophy UK and the SMA charities are also encouraging the SMC, the Scottish Government and NHS Scotland to introduce a reformed appraisal process for ultra-orphan medicines as quickly as possible. Meanwhile, applications for patients with Type 2 and 3 SMA are being made on a case-by-case basis by treating hospitals in Scotland.
Robert Meadowcroft, Chief Executive of Muscular Dystrophy UK, said:
“Spinraza brings hope to many families, and we welcome the news that it will be made available by NHS Scotland to children with SMA Type 1. But we won’t rest until all patients with SMA have access to this treatment, and it is now vital that robust data is provided by Biogen to support the argument for access for all people with SMA, as we turn our focus to encouraging the SMC to reassess the drug for other types.
“SMA is a devastating and cruel condition, and while it is not a cure, Spinraza can buy families more time. No parent should have to see their child gradually lose the ability to move, breathe and swallow, particularly while there is a treatment out there which could help.”
Spinraza proved so effective in a clinical trial for children with SMA Type 1 that the trial was stopped early so that all children affected could access the treatment. Treatment was delivered to children in Scotland via a temporary special compassionate access scheme, with the drug provided free by pharmaceutical manufacturer Biogen and the NHS in Scotland funding the costs of administering the treatment. At a special meeting earlier this year, families affected by the condition made a final plea to the SMC to approve Spinraza.
Dr Sheonad Macfarlane, Muscular Dystrophy UK’s Scottish Council chair, shared her family’s experience of living with SMA at the SMC’s PACE (Patient and Clinical Experts) meeting earlier this year. Her nine-year-old daughter, Eilidh, has SMA Type 2. She said:
“I understand the decision that has been made, and it is wonderful news for families of children with SMA Type 1; however, many others with the condition will miss out, and naturally this is a huge disappointment. It doesn’t end here, though, and we will keep fighting until all patients with SMA have access to this life-changing treatment.”
Amy Cameron, 30, from Alloa, Clackmannanshire, Scotland, whose son Zac, two, has SMA Type 1, and has been receiving Spinraza through a temporary special compassionate access scheme, said:
“Spinraza gives children with SMA the chance to reach amazing milestones that were never before possible. We are delighted that children like Zac will have access to this life-changing treatment, which has made such a difference to our lives. Since receiving Spinraza, Zac can swing his legs out when we hold him on our knee, and can also hold his head up when I pick him up. He’s never done this before – it’s a complete miracle. The effect Spinraza has on families cannot be underestimated, and it should be available to all who need it.”
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Notes to editors
About muscle-wasting conditions
- More than 70,000 children and adults in the UK have a muscle-wasting condition.
- Muscle-wasting conditions may be inherited or strike out of the blue. These conditions cause muscles to weaken and waste over time, leading to increasingly severe disability. Some affect the heart and muscles vital for breathing, cutting lives short.
Muscular Dystrophy UK
- Muscular Dystrophy UK is the charity for 70,000 children and adults living with muscle-wasting conditions. We provide vital information, advice and support to help people live as independently as possible. We accelerate progress in research and drive the campaign for access to emerging treatments.