Update on Nusinersen Expanded Access Programme in the UK

Update on progress towards an Expanded Access Programme of the ENDEAR trial of nusinersen which treats eligible infants with Spinal Muscular Atrophy Type 1  

Joint statement from The SMA Trust, SMA Support UK and Muscular Dystrophy UK. 

Families may recall reading the last update in September 2016 which was released by the Principal Investigators from the two trial centres at Newcastle Neuromuscular Centre and Great Ormond Street Hospital (GOSH).

The ENDEAR study results haven’t yet been fully analysed and there is only limited knowledge about the efficacy of nusinersen across the full spectrum of infants and children with SMA Type 1.  However, results have been sufficiently positive for Biogen to work on opening an Expanded Access Programme (EAP).

An Expanded Access Programme (EAP) is designed to provide access to treatment for eligible infants with SMA Type 1 who didn’t participate in a clinical trial. The programme is aimed at providing access prior to any European Medicines Agency approval.

On Wednesday 14 December, representatives of the two Centres met with Muscular Dystrophy UK, SMA Support UK, The SMA Trust and Biogen. The questions and answers below cover the discussions about the progress so far and the next steps, and are being publicised by the three charities on their websites and through their social media channels.

All involved with the discussions understand how frustrating and distressing the process can be for families. We are all doing all we can to move things forward and will continue to update you as soon as more information is available.

Have any decisions been made about which children will be on the EAP?

In countries with higher numbers of eligible infants, it is impossible to recruit all children at the same time. There is therefore ongoing discussion on how to prioritise the recruitment of children into the EAP. This discussion is held in collaboration with other countries in which similar issues are being faced; and with the local ethics committees at the sites involved. Factors that will be taken into account include the medical need based on the information available so far about the risks and benefits there have been to the children treated in the ENDEAR study. Therefore, this may involve prioritising babies likely to receive the greatest benefit from the treatment.  Other criteria that are being considered include the distance from individual centres/ travel; the severity of the condition and the age of the patient.

Where will the treatment be offered in the UK?

An EAP has opened in Belfast for those living in Northern Ireland as the number of infants currently eligible for treatment there are so small. GOSH and Newcastle are in active discussion as to the best way to open the EAP at their individual centres. A number of other UK centres have expressed interest in being involved and in the next few weeks there will be discussions with Biogen to see if this is possible. We will provide an update on this as soon as this information becomes available.

When will the EAP actually start at Newcastle and GOSH?

At the moment it is not possible to give an exact date when the EAP will begin at Newcastle and GOSH. The EAP could only start there once the children originally recruited to the randomised placebo part of the ENDEAR study had been transitioned to the nusinersen treatment. This process has now been completed so the process of initiating the EAP at those two centres is starting now.

How does a family get on to the programme?

Families who are resident in the UK may apply by contacting the coordinators in London: and in Newcastle: Families living in Northern Ireland may contact Belfast Children’s Hospital on 028 9032 9241 though we understand there is no capacity to enrol more infants in Belfast at the moment. Families can also contact Biogen by email: to obtain more information.

Can you tell us exactly what the treatment involves?

The drug needs to be administered using a lumbar puncture. In the first year there are 4 injections in the first 2 months, and a further 2 injections in the remaining 10 months. From the second year onwards it is anticipated children will require 3 lumbar punctures each year.

The lumbar punctures require special precautions and, especially in older children, will require either sedation or general anaesthetic. These are particularly complex procedures in children with SMA Type 1.

Are there any possible side effects / problems with the treatment we should be aware of?

The information available so far appears to indicate no drug related adverse event. However, as the drug requires repeated lumbar punctures, common problems relating to this procedure, such as headaches, have been reported in several of the children studied.

What if a child was on the EAP and became unwell at the time of one of their scheduled treatments – would they still be able to carry on with the programme?

The lumbar puncture is a complex procedure which can only be performed if children are well. If a child is too unwell, the lumbar puncture will need to be rescheduled.

Who will pay for the drug and any other costs to do with the treatment?

The drug will be made freely available by Biogen. The cost of the assessment and administration of the drug will be covered by the NHS and the local hospitals.

Would there be help with travel and accommodation costs if needed?

This will be the same as for any hospital appointment.

We know Biogen has applied to National Institute for Clinical Excellence (NICE) to begin the process of applying for the treatment to be approved in England. How does this fit in with the EAP and who gets involved in this licensing decision?

Before NICE can formally review the drug for approval in England, a decision on the drug’s licence must be taken by the European Medicines Agency (EMA). The EMA’s process for assessing the drug has just begun, and it is expected a decision will be reached later in 2017. In the meantime, NICE is currently conducting what is called a ‘scoping’ process. This 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 in January 2017. Muscular Dystrophy UK and representatives from SMA Support UK and The SMA Trust will be there.

The EAP therefore provides the most immediate prospect of access to the drug for patients meeting the criteria. However, in the long term it will be important to receive approval from the EMA and NICE.

What are the processes in Scotland, Wales and Northern Ireland?

In Scotland, decisions on approval after an EMA licence is granted are taken by the Scottish Medicines Consortium (SMC). Wales and Northern Ireland also have separate processes – although often these two countries follow the decisions made by NICE.

We have heard that the drug may soon be licenced in the US. Will that affect what happens in the UK?

No – the United States has a separate process for approving new treatments.

Is there any other way families can get the treatment now – either privately or by going to another country?

No – the treatment can only currently be accessed via the EAP in the UK.

What else is happening to help speed things up?

The EAP was discussed at Muscular Dystrophy UK’s Services Development Committee meeting in Parliament this month. MD UK has also highlighted the need for access to nusinersen with NHS England’s paediatric neurosciences clinical reference group (CRG). CRGs are groups of clinicians, commissioners, public health experts, patients and carers who use their specific knowledge and expertise to advise NHS England on the best ways that specialised services should be provided. Data will be presented to the group in January. At the next meeting in January, the group will also consider the multidisciplinary resources needed to support centres involved in the centres to deliver the treatment.

What can families do to help?

It is important that decision makers understand SMA Type 1 and nusinersen. If you would like to help to raise awareness of the issue with your MP – so that they can raise the issue on your behalf – please contact Peter Sutton at MD UK on


If you have any other questions, families can contact Biogen by email at:

Physicians can contact:

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 and social media. You can also contact us on 01789 801155 or email:

You can also contact SMA Support UK on 01789 267520 or email:

Leave a Reply

Your email address will not be published. Required fields are marked *