Newcastle MP ‘over the moon’ about Batten Disease decision

Newcastle North MP, Catherine McKinnell, has warmly welcomed today’s announcement by NHS England and the National Institute for Health & Care Excellence (NICE) that an innovative treatment for CLN2 Batten Disease will now be made available to patients on the NHS.

Hosting the Rich family in Parliament earlier this year

Catherine has fought extensively for access to Brineura on the health service on behalf of her young Throckley constituents, Nicole and Jessica Rich, who have been diagnosed with this rare, genetic condition, and have been receiving Brineura, an enzyme replacement therapy, at Great Ormond Street Hospital in London.

Licensed by the European Medicines Agency in 2017, children receive the treatment – also known as cerliponase alfa – by an infusion into the brain over four hours once a fortnight. However, clinical trials and the compassionate use programme that had been enabling the therapy to take place in England did not provide security of access to what is the first and only treatment for any form of Batten Disease, because the clinical trial was due to end next year, whilst the compassionate use programme could have been withdrawn at any time.

Today’s announcement means that Brineura will be accessed by patients with CLN2 Batten Disease as part of a ‘managed access agreement’, and will – unusually – be rolled out on the NHS ahead of NICE publishing its final guidance on the treatment later this year.

The Newcastle North MP’s campaign on this issue has included securing a meeting for the Rich family with Secretary of State for Health & Social Care, Matt Hancock; regularly raising the issue in Parliament (most recently here; here and here); and repeatedly lobbying Ministers, NICE, NHS England and the manufacturer of Brineura, BioMarin, for over a year.

Catherine said:

‘This has been a long, hard-fought campaign by the Rich family, which I’ve been pleased to support in any way I could as their local MP.

‘I am absolutely over the moon about this decision for Gail, Matthew, Jessica, Nicole and their brother Louis – and for all the other families affected by this cruel, debilitating disease.

‘To know that an effective treatment is available for your children – and to face them not being able to access it within just a few months, not to mention those families who have not been able to access it up to now – has been absolute agony for everyone involved and I couldn’t be happier about today’s announcement.

‘Hopefully, the Rich family will now be able to focus all their precious time and energy on their beautiful children, instead of having to fight for this drug.’

Letter from NHS England Chief Executive confirming today’s decision