European Commission approves nuisnersen to be marketed as Spinraza for those with 5qSMA which includes SMA Types 1, 2 and 3
We should now hear soon if there will be a Ministerial referral to NICE for appraisal for funding by the NHS.
This is the first treatment developed since the SMN1 gene was first identified 20 years ago and is the culmination of years of research.
Nusinersen aims to increase the production of SMN protein in the nerve cells. It achieved extremely positive results in clinical trials. Though trials were incomplete, there was sufficient evidence for nusinersen being safe and well tolerated, and encouraging evidence for its clinical effectiveness in children with infant onset SMA.
In the UK, the availability of the drug will now depend on a recommendation by the National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium and other authorities in the devolved nations to decide whether to fund the drug in England, Scotland, Wales and Northern Ireland. Availability may also depend on the treatment readiness of specialist centres.