We are now able to report that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the marketing approval of nusinersen, or Spinraza™ as it will now be called, for the treatment of 5q spinal muscular atrophy (childhood onset forms of SMA – Types 1,2 and 3) caused by a fault in the SMN1 gene. 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.
The CHMP opinion forms the basis for a European Commission decision on whether to grant a licence. It then usually takes around three months to formalise the licence.
In the UK, the availability of the drug will 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.
Please join us, MDUK and SMA Support UK in calling for a Ministerial referral to NICE to set this process in motion – and for NHS England to support delivery of the drug now to children with SMA Type 1 via the Expanded Access Programme. Here is a template letter.