Read the nusinersen Q&A
THE FIRST DRUG/TREATMENT FOR SMA – NUSINERSEN (SPINRAZA) APPROVED BY FDA IN THE US
Just before Christmas, the United States Food and Drug Administration (FDA) announced that it has approved nusinersen as a treatment for SMA. Spinraza is the brand name for the drug, nusinersen. This approval is for a broad licence, meaning that Spinraza can be marketed in the US for children and adults with SMA types 1-3.
Although the approval process in Europe is separate to the US, it’s a major step forward that the drug may soon be available in clinics in the US.
The European Medicines Agency (EMA) is responsible for approving potential treatments in Europe and the UK. Nusinersen’s manufacturer, Biogen, has applied to the EMA for a broad licence. The EMA is currently reviewing this and is expected to reach a decision later this year.
We hope that the outcome will be similar to that in the US and that a broad licence will be approved. Alongside Muscular Dystrophy UK and SMA Support UK, we are writing to the EMA urging a broad approval and for them to accelerate their assessment of the drug.
If the EMA approves nusinersen, it will be up to 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. This would usually be in line with the terms of the licence set out by the EMA.
NICE has already started what is called a ‘scoping’ process, which means it is gathering evidence about SMA and nusinersen, to help decide whether to review the drug should the EMA grant Biogen a licence. An initial meeting to consider this will take place this month. The SMA Trust and representatives from Muscular Dystrophy UK and SMA Support UK will be there. Together, we will be advocating for a review that will consider broad approval for all types of SMA. If a review does go ahead there will then be an opportunity for families to have their say.
Read the FDA Press Release
For more information:
- Watch a video that explains what SMA is and how nusinersen works
- Is nusinersen appropriate for my child? Read our Q&A
- Find out about the nusinersen Expanded Access Programme (EAP) in the UK and read the full Q&A about the progress so far and the next steps.
- Read about how The SMA Trust is working hard to obtain approval for nusinersen and details of the NICE Scoping Workshop that took place on January 13th.
- Get involved in the campaign to bring Spinraza to the UK
The SMA Trust will continue to work with MD UK, SMA Support UK, NICE, clinicians and Biogen in order to move things forward. We will keep you informed of progress via our website, email and social media.
We will continue to do all we can to move the process forward, keep you updated and ensure that your voices are heard.