NHS England Announces Access to Nusinersen for all with SMA Types 1, 2, 3a and 3b

15 May 2019

At last the SMA Community has the answer it has been asking for since NICE started its appraisal almost 17 months ago. The treatment, also known as SpinrazaTM, will be made available by Biogen immediately to the youngest and most severely-affected infants (SMA Type 1). The NHS will then begin to provide nusinersen to those with SMA Types 2 and 3 shortly after NICE’s full guidance is published on 26th June, and once the services to deliver them are established. It will also be available to pre-symptomatic siblings who, based on their genetics, are very likely to develop SMA. This includes, for example, newborns who are siblings of children already diagnosed with SMA and who therefore have a genetic test for the condition.

Starting and stopping criteria will be clearly set out in a Managed Access Agreement (MAA) which will now be finalised. This will clarify the starting criteria for treatment for anyone who has shown symptoms of SMA before the age of 18. The MAA will also define any stopping criteria. Treatment should therefore be available to adults who meet the starting criteria, but services will need to be established. At this stage we have no details of the criteria except to comment that these will be agreed with clinicians and we expect them to be fair and reasonable.

There have been no trials of nusinersen with anyone who has SMA Type 4 which is why adults with this adult onset form are not included in the agreement.

This announcement brings England in line with Scotland. We expect Wales and Northern Ireland to adopt very similar agreements.

Our thanks to NICE, NHS England and Biogen for this great news. Our thanks too to the patient and clinical representatives who spoke up at the NICE committee meetings; to all who made submissions to NICE, wrote to MPs, NICE, NHS England and Biogen; spoke up at the MP drop-ins; told their stories to the media and campaigned in so many different ways. Thanks to the MPs who supported their constituents by writing letters and asking questions and the clinicians who continued to push for access. Thanks to MDUK and TreatSMA for working so hard on this journey. The clinical evidence was there; our voices were finally heard.

We are only sorry that it took so long when time matters so much: for the families with infants with SMA Type 1 who have had no access to treatment since 1st November 2018; for families and adults who have desperately wanted to have the opportunity to see what potential this treatment might have for them; for the clinicians who have been so frustrated by their lack of power to offer it.

We will now do all we can to support the smooth implementation of the Managed Access Agreement (MAA) and will bring you any updates as soon as we have them. We will also continue to work to advocate for change to the NICE process for appraising access to orphan medicines so that future treatments are more rapidly and appropriately assessed. Find out more here

NHS England’s full press release

Press Release from Biogen in USA

Community Statement from BIOGEN UK