Your MP

Dear

I am writing to you regarding access tonusinersen (brand name Spinraza): the first and only treatment for Spinal Muscular Atrophy (SMA).On 1st June, nusinersen was approved by the European Commission for marketing as a treatment for those with 5qSMA which refers to a fault on Chromosome 5.It is strongly supported by clinicians.

About SMA

SMA is a rare inherited neuromuscular condition affecting the lower motor neurons. It may affect crawling and walking ability, arm, hand, head and neck movement, breathing and swallowing. There are four main types of 5qSMA, three of which have childhood onset and are called SMA Types 1, 2 and 3. The effects of the condition vary but,for most children with SMA Type 1 - the most severe type - life expectancy without intervention is less than two years.

About nusinersen

Nusinersen is manufactured by the pharmaceutical company Biogen and is marketed as Spinraza. Clinical trials with infants and children with SMA Types 1,2 and 3 have shown significant improvement in children’s motor function, allowing them to achieve or maintain physical milestones that they would never have reached without treatment, and to survive longer than expected considering the typical course of the condition.

Administration of the drug requires a lumbar puncture injection into the spinal cord. 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 procedure is not dissimilar to those already routinely performed by the NHS.

Expanded Access Programme for eligible infants with SMA Type 1

You may already be aware that in autumn 2016, Biogen opened a global ‘compassionate use’ or Expanded Access Programme (EAP) for eligible infants affected by SMA Type 1. Biogen provides the drug free for the treated child’s lifetime however, the administering country must fund the other costs of treatment – staff, clinic / theatre and any other related costs.

Though the EAP has been successfully launched in the rest of Europe (some 350 children on the programme) , in the UK, especially England, we are lagging well behind (we understand that at the beginning of June there was a total of only 11 children on the programme in England). In many areas of the country, despite the efforts of clinicians, lack of capacity at specialist centres appears to be the main reason for this,along with lack of funding from NHS England for ancillary costs. This hascreated a post code lottery and is causing great distress to families. This is especially concerning given trial findings which strongly indicate the earlier the treatment is delivered the more likely a positive outcome.

We know of desperate families who have fundraised and have been travelling to Paris with their sick children for treatment. One such family has been told that now the European Commission has approved the drug the last treatment they can access there is likely to be in August this year. We know of other families with no local access to the programme who are unable to travel or raise funds who have been frantically writing to all Trusts in the UK to see if their child can be accepted on the programme in another area.

I am asking you to contact Simon Stevens at NHS England to urgeNHSE to reach an agreement with the drug company Biogen and relevant NHS Trusts without delayso that key centres are supported to deliver the drug to all eligible children with SMA Type 1 now.

Longer Term Access to nusinersen for all those with 5qSMA

Now that the European Commission has given marketing authority to the treatment, I would also be grateful if you would write to Andrew Dillon at NICE to ask that urgent steps are taken to review whether to recommend the drug for funding by the NHS for those affected by all types of 5qSMA.

Thank you for taking the time to consider theserequests –I very much hope you will be able to help progress these urgent issues.

Yours sincerely,