Treatment which can prolong the lives of children with a rare genetic condition will be made available on the NHS.
Nusinersen, also called Spinraza and made by Biogen, is the first treatment that targets the underlying cause of spinal muscular atrophy (SMA).
The number of children and adults in England and Wales living with SMA
NHS England said it has successfully negotiated a deal with Biogen, describing it as “one of the most comprehensive deals in the world”.
SMA affects the nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing.
Where it develops in babies and toddlers, it can significantly reduce life expectancy.
Between 600 and 1,200 children and adults are currently living with the condition in England and Wales.
While not a cure, trials have shown that nusinersen can slow the effects of SMA in some cases, allowing babies and toddlers to develop stronger muscles and survive for longer without breathing support.
It is injected directly into the spine and is a lifelong treatment.
The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits
Simon Stevens, NHS England chief executive, said: “This promising treatment has the potential to be life-changing for children and their families.
“The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits.
“This latest deal coming on the heels of a number of other recent successful negotiations demonstrates that there is no reason for other companies not to show equivalent flexibility in order to benefit NHS patients, taxpayers and indeed themselves.”
NICE has previously not been able to recommend nusinersen for routine use because of uncertainties over its long-term effectiveness and its high cost.
The agreement that has been reached between NHS England and Biogen is known as a managed access agreement, meaning that the NHS will fund treatment for a time-limited period, allowing further data to be collected on its effectiveness.
This paves the way for NICE to formally approve the treatment for use with eligible patients through publishing final guidance.
The treatment will be made available to the youngest and most severely-affected (SMA type 1) patients immediately by Biogen, with NHS England offering funding on NICE’s publication of final guidance.
For older babies, children and young adults with less severe symptoms (SMA types 2 and 3), the NHS will begin to provide nusinursen shortly after NICE’s guidance is published, once the services to deliver them are established.
NHS England said this is in line with the approach for making other brand new treatments available in a fair way for patients across the country and is not expected to take more than a few weeks.
All relevant SMA patients (SMA 1,2,3a and 3b), including adults and siblings who are yet to show symptoms, will be able to benefit from this treatment, NHS England said.