Medical research

Spinraza (nusinersen)

Read the nusinersen Q&A


On 21st April, the Committee for Medicinal Products for Human Use (CHMP) announced that it has recommended Biogen be granted a license for 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 will used for children and adults with SMA types 1-3.

The European Commission will now review the CHMP’s recommendation. This process will take about 3 months. If and when the European Commission grants Biogen a license, 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 European Commission.

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 took place in January, attended by The SMA Trust and representatives from Muscular Dystrophy UK and SMA Support UK. Together, we advocated 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.

On 5th April 2017, the North Star / SMA-REACH UK group of seventy clinicians, physiotherapists, study coordinators, industry and advocacy groups’ representatives, (including SMA Support UK who represented both SMA advocacy organisations), sent an open letter, signed on behalf of the group by Professor Francesco Muntoni, setting out their position and recommendations to the National Institute for Health and Care Excellence (NICE), NHS England’s Care and Clinical Reference Groups (CRGs), NHS Trusts and Biogen.

You can read the letter and see the full list of the signatories here and read SMA Support UK’s explanation of the letter’s content.

Read the CHMP Press Release

For more information:

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.