First treatment available for spinal muscular atrophy
Author: Jo Carlowe
Children with the rare muscle-wasting disorder spinal muscular atrophy are to benefit from a promising new treatment 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).
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.
Working with the National Institute of Health and Care Excellence (NICE), NHS England has successfully negotiated a deal with Biogen, meaning that this treatment will be available for those whose clinicians think they would benefit.
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.
NHS England and Biogen reached 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. 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.
This is one of the most comprehensive deals in the world, meaning 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.