nusinersen (Spinraza) – a milestone but not the end game


The news from the US of the FDA’s approval of nusinersen (now marketed as Spinraza in the US), the first treatment for SMA, has changed the SMA landscape for ever.  Accelerated approval is also being sought from The European Medicines Agency (EMA) for a European licence and then it will be up to individual countries to appraise the evidence and decide whether to fund the drug.

Those who have followed recent updates will have seen that The SMA Trust is actively involved in all aspects of this process, working closely with the other charities, clinicians, Biogen, EMA and health technology appraisal bodies such as NICE.  We are doing everything we can and fervently hope that nusinersen (Spinraza™) will be available to all those who might benefit as quickly as possible, whilst accepting and working within the necessary processes and timelines for the approval of any new drug.

In the meantime, our other work continues and is just as important as ever.  nusinersen (Spinraza™) is a milestone but not the end game.   Other gene therapy treatments are in the pipeline, together with alternative ways of administering them;  and there are also encouraging results coming out of research into the development of potential treatments for other aspects of SMA, which may well be used in combination with drugs like nusinersen.

The SMA Trust is involved in all these areas, both through the UK SMA Research Consortium (which we are funding at a cost of £1.3 million) and through our contribution to SMA Europe  projects.  In addition we continue to fund SMA REACH, a clinical network which collects and analyses data from SMA patients in a way that can be used for all future UK clinical trials.  We are also working to increase the number and geographical spread of trial sites so that more people affected by SMA in the UK will have the opportunity to take part.

We remain dedicated to finding a cure and treatments for SMA and will do anything in our power to achieve that goal.  However, we are totally dependent on voluntary donations and desperately need your continued support to ensure we can carry on doing all the things that are so vital to the maintenance of current momentum and progress.

Find out how The SMA Trust is working hard to bring Spinraza to the UK

 Find out about all our SMA research underway

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