Batten Disease Family Association

‼️ In response to the decision from NICE to extend the MAA for Brineura by six months, the BDFA is urgently calling for an agreement for permanent access for patients with CLN2 ‼️ The National Institute for Health and Care Excellence (NICE) has extended the Managed Access Agreement (MAA) for cerliponase alfa (Brineura®) for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) by six months so that it can remain available through the NHS for that period and to allow the manufacturer (BioMarin) and NHS England (NHSE) to negotiate a long-term commercial agreement. Read full statement from NICE here https://lnkd.in/e7Sw8Gvn The Batten Disease Family Association CIO (BDFA) expresses deep dismay that after five years of the MAA, characterised by rigorous clinical assessments and burdensome data collection for families affected by CLN2, and two evaluation meetings held on 12th June and 5th September 2024, NICE has failed to issue any guidance about the outcome of its evaluations. This raises serious concerns about the efficacy of the NICE process, the purpose of the MAA, and the overall impact of the data collection and clinical assessments. Given the circumstances, while an MAA extension may be helpful, we fear it may merely prolong the agonising wait for families seeking clarity about their children’s futures. The CLN2 community demands assurance that this extension is not a delaying tactic and that NICE, BioMarin, and NHS England will use this time effectively to reach a permanent access agreement for Brineura® as a matter of urgency. Moreover, with data collection no longer required, we insist that rigorous clinical assessments also cease immediately. Brineura® is a groundbreaking treatment and its availability over the last five years under an MAA between NHSE and BioMarin has been a lifeline to families who, before then, had no other treatment options. There is no cure for this neurodegenerative disease and without treatment, children living with CLN2 risk deteriorating very rapidly. Withdrawing access from the NHS treatment could lead to a situation where the future of a child’s access to Brineura® is uncertain, and where one child with Batten disease has access and another does not. Liz Brownnutt, Chief Executive of the BDFA: “We do not want the MAA extension to lessen any sense of urgency for the parties involved. Coming to a long-term agreement to allow continued funding of this life-saving treatment will provide security to the patients and families who so desperately need it and will put an end to their agonising wait. We will not stop fighting until all eligible CLN2 patients in the UK are able to access Brineura®.” Click this link to read the BDFA’s press release in full on their website https://lnkd.in/eTTT8r7R

❗ Breaking News❗ The BDFA is delighted to share that Tern Therapeutics, a biotechnology company based in the USA have entered into a global licensing agreement with REGENXBIO Inc. for RGX-381 and RGX-181. The company’s Chief Executive Officer, Alex Bailey and Chief Medical Officer, Christina Ohnsman were both instrumental to the development of the CLN2 programs in their previous roles at REGENXBIO. They will be using their experience and unique capabilities to initially focus on continuing the RGX-381 clinical trial in the UK at GOSH through to completion. The BDFA will be working in close contact with Tern Therapeutics, also continuing to work with REGENXBIO, and we are thrilled with the news of this licensing agreement, which will bring hope to the CLN2 community. We are pleased to share the company’s full press release, which contains a quote from Liz Brownnutt CEO of the BDFA, and a letter to the community from REGENXBIO. Tern Press Release: https://lnkd.in/etUqFUDY Regenxbio Letter to Community: https://lnkd.in/ewAcuXt7 Please get in touch with Liz if you have any questions - lizbrownnutt@bdfa-uk.org.uk 07745210212

🌎 Global Update - Clinical Trial in CLN5 disease update from Neurogene Inc. 🌎 Neurogene Inc. announced that its Phase 1/2 Trial of NGN-101 Gene Therapy for the treatment of CLN5 Batten disease is now fully enrolled (n=6), with plans to provide interim clinical data and a regulatory update in the first quarter of 2025. You can read more on our website, link below https://lnkd.in/epMqxpvn

🔶 Batten Disease Global Research Initiative Grants 🔶 Now Open for Expressions of Interest until AUGUST 30th   Together, the BDFA, with our Global Research Initiative (GRI) partners, is delighted to announce the inaugural GRI Grant Round is now inviting Expressions of Interest.    We seek to support the most promising research ideas worldwide that address key research questions and areas of unmet need in Batten disease. Submissions CLOSE AUGUST 30. To find out more about the Program and how to apply, please visit: https://lnkd.in/ecX2DiNs   #BattenAdvocatesForACure #GlobalResearchInitiative #Research #BattenDisease   Please contact Jo Nightingale if you have any queries, joannanightingale@bdfa-uk.org.uk

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Today is International Batten Disease Awareness Day. Batten disease is a fatal, genetically inherited disorder affecting lysosomes which recycle material inside cells. Children are born with no signs there is anything wrong. Early in childhood their learning stagnates and regresses. They suffer from seizures, blindness and dementia. There is NO cure. Batten disease is ultra rare affecting about 1 in 100,000 live births. There are less than 200 children and young adults affected in this country. There are 13 genes that cause different types of Batten disease. Only one of these has a treatment to slow down progression - Brineura for CLN2 Batten disease. Please help us raise awareness by telling just one person about it today and if you can, please make a donation. Thank you.

🔸 IT’S INTERNATIONAL BATTEN DISEASE AWARENESS DAY 2024 🔸 Today is all about raising awareness of Batten disease with the global Batten community. Please join us by wearing orange and telling just one person about Batten disease today. Whilst we love Awareness Day to be a celebration of the incredible people in our community, we know this day can also be also be difficult for families affected by Batten disease and we send our love to you all.

🔸 International Batten Disease Awareness Day takes place on Sunday 9th June 2024 🔸 The Batten Disease Family Association (BDFA) are thrilled to have launched a ‘Text to Donate’ Awareness Campaign which is now LIVE! We would like to say a huge thank you to Beefy's Charity Foundation and Lord Ian 'Beefy' Botham for his continued support and for encouraging you to get involved with this year’s International Batten Disease Awareness Day. Details of how to donate are on the video below! Thank you and Together We WILL Make a Difference.

🔸🔸6 days to go until International Batten Disease Awareness Day 2024🔸🔸 It’s officially Batten Disease Awareness Week and the BDFA are thrilled to announce we have launched a ‘Text to Donate’ Awareness Campaign! Our Text to Donate campaign is live from TODAY🧡🧡 Details of how to donate are on the poster below. Please share this post and help us raise vital funds along with invaluable awareness. Thank you!!✨🧡

We’re endorsing @kidscharity new report, which reveals the huge gap in support faced by young people with SEND as they move from children’s to adults’ services. Read and share the report 👇 https://lnkd.in/emTab_ii