Medical research

Access to Spinraza, the first treatment for SMA

On 21st April, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) announced that it had recommended Biogen be granted a license for nusinersen as a treatment for SMA. Spinraza is the brand name for the drug, nusinersen. This approval is for a broad licence, meaning that Spinraza will be used for children and adults with SMA types 1-3.

The European Commission reviewed the CHMP’s recommendation and on 1st June, announced that it had granted Biogen marketing authorisation.

What does this mean for the UK?

The National Institute for Health and Care Excellence (NICE), NHS England, the Scottish Medicines Consortium and other authorities in the devolved nations will now decide whether to fund the drug in England, Scotland, Wales and Northern Ireland. This is usually in line with the terms of the licence set out by the European Commission.

NICE started what is called a ‘scoping’ process, gathering evidence about SMA and nusinersen, to help decide whether to review the drug should EMA grant Biogen a licence. An initial meeting to consider this took place in January, which was attended by representatives of The SMA Trust, Muscular Dystrophy UK and SMA Support UK. Together, we advocated for a review that will consider broad approval for all types of SMA. If a review does go ahead there will then be an opportunity for families to have their say.

What are we doing to ensure access in the UK?

  • Position statement to NICE, NHS England’s CRGs, NHS Trusts & Biogen

On 5th April 2017, the North Star/ SMA-REACH UK group of seventy clinicians, physiotherapists, study coordinators, industry and advocacy groups’ representatives, (including SMA Support UK who represented both SMA advocacy organisations), sent an open letter, signed on behalf of the group by Professor Francesco Muntoni, setting out their position and recommendations to the National Institute for Health and Care Excellence (NICE), NHS England’s Care and Clinical Reference Groups (CRGs), NHS Trusts and Biogen.

You can read the letter and see the full list of the signatories and read SMA Support UK’s explanation of the letter’s content.

  • All Party Parliamentary Group for muscular dystrophy

MPs and Peers in the All Party Parliamentary Group (APPG) for Muscular Dystrophy held a meeting on 18th July dedicated to focusing on the key points relating to access to Spinraza.
At the meeting, chaired by Mary Glindon MP, representatives from NHS England, Biogen, Great Ormond Street Hospital and leading neuromuscular consultants, shared their views in a Q&A discussion on the current provision of Spinraza for infants with SMA Type 1 through the Expanded Access Programme (EAP) set up by Biogen and the next stages of the assessment process for Spinraza.

Representatives of Muscular Dystrophy UK, SMA Support UK, The SMA Trust and Treat SMA were all at the meeting to support individuals and families affected by SMA. Several testimonies were shared which made a powerful impression on MPs and Peers.

The main points from the Q&A included:

NHS England is exploring the possibility of an interim commissioning policy for the most severely affected;

Referral from the Health Minister for NICE to assess Spinraza is expected in the next few weeks;

Update from Biogen on active sites across the UK for the EAP and comparisons with access in other European countries;

Action taken by Great Ormond Street Hospital to support the delivery of the EAP;

Lack of NHS capacity and bottlenecks delaying access to Spinraza through the EAP.

The SMA Trust, alongside the other SMA charities, will continue to press for Spinraza to be made available to as broad a spectrum of SMA patients as possible.

Join us!

Access to Spinraza the first treatment for SMATo keep the issue in the spotlight, we are asking families to write to their representatives in all four UK Parliaments:

For more information

Access to Spinraza the first treatment for SMAAbout Spinraza

The Expanded Access Programme (EAP)