Latest news with #Brineura
BBC News
2 days ago
- Health
- BBC News
Mum 'reassured' over Batten disease drug access decision
"To know that he will have that treatment now for the rest of his life is incredible, it's so reassuring."Emily's four-year-old son, Max, from Spinkhill, near Killarmarsh in Derbyshire, has CLN2 Batten disease, a rare degenerative genetic disorder that causes a decline in a child's ability to walk, speak and is symptom-free and has been receiving Brineura, the only approved treatment that slows the condition's progress, since he was access to the drug, which costs over £500,000 per patient per year, had been due to expire at the end of May, however an agreement was reached allowing existing patients to continue to have it. However, it has not been recommended for future patients diagnosed beyond the end of 2025 "due to its high price and the limited evidence of long-term effectiveness", said the National Institute for Health and Care Excellence (NICE). Max goes to Manchester Children's Hospital every two weeks to receive treatment."He has to stay on the bed for four hours, but because he's had this treatment since he was a baby he doesn't really know any different so he likes it," Emily said."He says he's 'going to get his medicine' and sees the doctors."His dad, James, said Max receiving the treatment from a young age "has been absolutely key in the happy little boy that you see".He added: "He's progressing amazingly well in terms of his abilities."He's still symptom-free in terms of Batten disease, so it's a real success for the drug." Max's older sister, Holly, also has Batten disease and had been receiving Brineura as part of her the six-year-old was diagnosed later in life and the family felt she was not getting as much benefit from the drug as her symptoms increased and agreed it should be said: "Holly was on the treatment for around 12 months, but in that time her condition progressed and she started to lose more and more of her abilities."Within the space of a year, she'd gone from being a relatively normal and happy four-year-old to not being able to walk, losing all of her speech and losing her ability to eat and swallowing normally." Emily and James's youngest child, four-month-old Rory, was born after the couple underwent in vitro fertilisation were screened in advance to check if the condition might be passed on again and, as a result, Rory does not have Batten said: "We were lucky to get four embryos that were unaffected and Rory was the first one that we transferred, so he's a healthy baby."On the decision to stop the use of Brineura for children not yet diagnosed with Batten disease, NICE said: "This committee took into account the condition's rarity, severity and the effect of cerliponase alfa [the drug marketed as Brineura] on quality and length of life."But using the proposed price of the medicine, the most likely-cost effectiveness estimate is not within what NICE considers an acceptable use of NHS resources."So, cerliponase alfa is not recommended."In response, the Batten Disease Family Association said: "Whilst this is naturally disappointing, it is important to note that this is not NICE's final guidance on the future of Brineura on the NHS."The NICE committee will meet again in July to consider evidence and consultation feedback in relation to whether patients not currently diagnosed with Batten disease could receive the drug in future.
Yahoo
18-05-2025
- Health
- Yahoo
Mum's relief over 'brutally unfair' drug access
A Coventry mother is "massively relieved" after being told her daughter will now have access to a life-enhancing drug indefinitely. Phoebe's six-year-old daughter Flory lives with CLN2 Batten disease, a rare and untreatable genetic disorder affecting about 40 children in the UK. After months of uncertainty, health bosses this week revealed those already taking or soon to start treatment could continue to have it beyond the end of May. But Phoebe believes it is also "brutally unfair" not all young people affected will benefit, and has vowed to keep fighting so everyone can access the drug. CLN2 Batten disease causes a rapid decline in a child's ability to walk, talk and see, with an average life expectancy of about 10 years. Drug Brineura is the only approved treatment that slows the condition's progress, but NHS access was due to end in May. The National Institute for Health and Care Excellence (NICE) revealed on Thursday NHS England (NHSE) had come to a new agreement with its maker, BioMarin, regarding existing patients. But it costs about £500,000 per child per year, and is not being recommended for future patients "due to its high price and the limited evidence of long-term effectiveness". After she had receiving confirmation Flory's fortnightly treatment would continue, Phoebe said: "Families will see our children living longer with a better quality of life, while being completely hopeless." She said getting the drug on the NHS had been "absolutely monumental" for Flory, and she believed her daughter was still walking, climbing and eating thanks to it. "To know that she's going to have access to this indefinitely... is everything," she said. "This treatment completely changes the trajectory of her life. Before, her entire life was uncertain." But while it is a massive relief for her family, she is determined to keep advocating for others. Phoebe urged decision makers to do the right thing, adding they were "halfway there". "Every child should be able to access this treatment," she said. "It is is life-changing. To offer it some and not others seems brutally unfair." NICE said it would keep working with NHSE and BioMarin towards a long-term deal for all patients after a managed access agreement ended in December. "We know this is not entirely the news people in the Batten Disease community were hoping for," said NICE director of medicines evaluation Helen Knight. "However, this is not the end of the story. We will continue to work with all parties towards a solution." The Batten Disease Family Association said it was pleased with the agreement, but would continue to push for Brineura to be accessible for all children. Follow BBC Coventry & Warwickshire on BBC Sounds, Facebook, X and Instagram. Child with rare disease will still get vital drug Relief as treatment for rare condition is extended Mothers rally and urge action over access to drug National Institute for Health and Care Excellence
BBC News
18-05-2025
- Health
- BBC News
Coventry mum's relief over 'brutally unfair' drug access
A Coventry mother is "massively relieved" after being told her daughter will now have access to a life-enhancing drug indefinitely. Phoebe's six-year-old daughter Flory lives with CLN2 Batten disease, a rare and untreatable genetic disorder affecting about 40 children in the UK. After months of uncertainty, health bosses this week revealed those already taking or soon to start treatment could continue to have it beyond the end of Phoebe believes it is also "brutally unfair" not all young people affected will benefit, and has vowed to keep fighting so everyone can access the drug. CLN2 Batten disease causes a rapid decline in a child's ability to walk, talk and see, with an average life expectancy of about 10 years. Drug Brineura is the only approved treatment that slows the condition's progress, but NHS access was due to end in May. The National Institute for Health and Care Excellence (NICE) revealed on Thursday NHS England (NHSE) had come to a new agreement with its maker, BioMarin, regarding existing it costs about £500,000 per child per year, and is not being recommended for future patients "due to its high price and the limited evidence of long-term effectiveness". After she had receiving confirmation Flory's fortnightly treatment would continue, Phoebe said: "Families will see our children living longer with a better quality of life, while being completely hopeless." She said getting the drug on the NHS had been "absolutely monumental" for Flory, and she believed her daughter was still walking, climbing and eating thanks to it. "To know that she's going to have access to this indefinitely... is everything," she said. "This treatment completely changes the trajectory of her life. Before, her entire life was uncertain." But while it is a massive relief for her family, she is determined to keep advocating for others. Phoebe urged decision makers to do the right thing, adding they were "halfway there". "Every child should be able to access this treatment," she said. "It is is life-changing. To offer it some and not others seems brutally unfair." NICE said it would keep working with NHSE and BioMarin towards a long-term deal for all patients after a managed access agreement ended in December. "We know this is not entirely the news people in the Batten Disease community were hoping for," said NICE director of medicines evaluation Helen Knight."However, this is not the end of the story. We will continue to work with all parties towards a solution."The Batten Disease Family Association said it was pleased with the agreement, but would continue to push for Brineura to be accessible for all children. Follow BBC Coventry & Warwickshire on BBC Sounds, Facebook, X and Instagram.
BBC News
16-05-2025
- Health
- BBC News
Swindon girl with Batten Disease will continue to get vital drug
A family has been told their child will continue to get access to a drug which she began taking as part of a trial, and is helping to keep her Addy Clarke, from Swindon, has Batten Disease - a rare degenerative disease that has no agreement has been reached that existing patients will continue to have the drug Brineura, which costs £500,000 per patient per year, via the it has not been recommended for future patients "due to its high price and the limited evidence of long-term effectiveness", said the National Institute for Health and Care Excellence (NICE). Addy's mother Hayley Clarke said she is "absolutely thrilled and relieved and so thankful" at the news but wants to get access to the drug for other children. "There's still another half a fight to go to get it secured for all children, future children most importantly," she said the family could have lost Addy by now, that she should be blind, but her eyesight has been preserved and she can still make herself of the main symptoms of Batten Disease is childhood dementia. "We're just so thankful for the time that this drug is giving us with her," said Hayley, who also told the BBC about her daughter's passions for lions and swimming."I think I'm still processing that it's a yes for our daughter," she added."[I'm] almost trying to be really guarded with hope because we've had too much shattered hope along the way."Hayley said the family has had a lot of support from people around them as well as local hospice charities and the council. The family went with other with members with Batten Disease to Westminster to ask for continued access to guidance from NICE did not recommend using it for future patients due to cost and long-term effectiveness, but said it had reached a deal with the NHS and manufacturer BioMarin for those already using it and anyone signed up by the end of 2025. Helen Knight, director of medicines evaluation at NICE said: "We know this is not entirely the news people in the Batten Disease community were hoping for. However, this is not the end of the story. We will continue to work with all parties towards a solution."The charity Batten Disease Family Association said it was pleased with the agreement, but said as the NICE guidance is a draft and not final, it will continue to push to make Brineura accessible to all children who need it.
Yahoo
16-05-2025
- Health
- Yahoo
Relief as treatment for rare condition is extended
A mother says she and her family can "live again" after access to her son's life-enhancing treatment for a rare genetic disorder was extended. NHS access to the drug Brineura, which slows the progress of Batten disease, was set to end this month but NICE and NHS England have now come to new agreement with the drug's maker. One of those who receives it is Isaac, eight, who has CLN2 Batten disease, which was diagnosed in August 2021. His mother Aimee Tilley, from Kettering in Northamptonshire, said: "We know it's not a cure, we still see regression, but it's a huge amount slower, so he's gaining years, not just days or weeks." Batten disease, a rare genetic disorder, causes a rapid decline in a child's ability to walk, talk and see, and is estimated to affect about 40 children in the UK - with an average life expectancy of about 10 years. Brineura is the only approved treatment that slows the condition's progress. The new agreement will mean those on the drug, and those who start the treatment before the end of the year, can receive it on a permanent basis. Ms Tilley said: "We are extremely relieved that Isaac is going to continue to have this treatment. "This black cloud that we've had hanging over us has gone. We feel like we can live again." NICE said it and NHS England would continue to work with BioMarin, which makes the drug, on "a solution to secure access to all future patients but at the moment the treatment is not considered cost effective". Ms Tilley says her family "will not stop fighting for the children of the future". She said: "They deserve it just as much as the children now and we have won this battle, but we will win the war." Ms Tilley said Isaac was "having seizures, losing his mobility, he can still walk with a walker or walk holding our hands [and] he has now gone blind". But, she added: "He's happy. He still enjoys theme parks, going horse riding and he still does a lot of things that children of his age can do we just have to adapt them for him." Helen Knight, director of medicines evaluation at NICE, said she was "pleased" an agreement had been reached. She added that NICE and NHS England remained "committed to working with the company to try to reach a long-term deal that will give access to [Brineura] to all eligible people" after December. Follow Northamptonshire news on BBC Sounds, Facebook, Instagram and X. Threat to son's treatment for rare disease 'agony' Mothers rally and urge action over access to drug NICE NHS England Batten Disease Family Association (BDFA)
