SMA Trust Spinraza update

SMA Trust Spinraza updateWe’re delighted with the news of the FDA’s approval of Spinraza in the US (as we reported just before Christmas) and, like everyone else, very much hope for EMA approval in a few month’s time, followed by NICE and other bodies involved in approving new drugs in the UK .

We’ve been working closely with EMA for a while, culminating in a recent 1-day workshop organised by EMA, TREAT-NMD and us through our membership of SMA Europe (Joanna Mitchell is a board member and was on the workshop steering group). The workshop helped to further inform senior EMA officials about the particular characteristics and impact of SMA, as well as the difference that future treatments could make to the lives of people affected by SMA.

We are also working closely with NICE on an initial scoping exercise to look at the best way for Spinraza to be appraised if and when it receives an EMA licence. We submitted a response as part of their consultation at the end of last year and will be attending a stakeholder workshop later this month. If and when EMA approval is given, we will also be dealing with the Scottish Medicines Consortium and equivalent bodies in Wales and Northern Ireland and will obviously do everything possible to convince them of the vital importance of approving Spinraza.

We also continue to work closely with Biogen at both European and UK level and have conference calls scheduled with all parties this week.

We are aware of the urgent need for accurate information and will be providing regular updates from now on as things develop on the web page dedicated to nusinersen/Spinraza.

If you have any questions in the meantime please contact us.

As with the statement and Q & A on the Expanded Access Programme, we will continue to collaborate closely with SMA Support UK and Muscular Dystrophy UK to ensure the SMA community is represented by the strongest and most consistent voice possible.

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