Coventry and Doncaster mums rally in London and urge action over access to Batten disease drugdrug

The mother of a six-year-old girl with a rare neurodegenerative disease has urged health bosses to “do the right thing” amid fears over the future of the medication she takes.

Flory, from Coventry, has CLN2 Batten disease, an incurable genetic disorder and Brineura is the only approved treatment that slows the condition’s progress.

Dozens of campaigners were in Westminster on Wednesday after being told NHS access to the drug was set to end in May.

Health officials claim “constructive” talks are being held over continued access to it but Flory’s mother, Phoebe, said: “We absolutely cannot let this drug be taken away.”

“We need to do the right thing here, there are 40 children who are dependant on this treatment to survive, to live, for a quality of life.”

The disease 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.

Getting Brineura on the NHS had been “absolutely monumental” for Flory, according to Phoebe, who added she was still walking, climbing and eating due to the drug.

Another parent, Anna, from Doncaster, was also at the rally and said removing the medicine would be “absolutely devastating” for her five-year-old daughter Beatrice, known as Bee.

“She would decline rapidly and we need Brineura to keep her being Bee and doing all the things…she’s loving life at the minute. She deserves a chance,” she said.

Anna told the BBC her daughter had an infusion of the drug every fortnight which “keeps her on her feet and talking”.

“She’s reading books, she’s swapping them at school so her life is the same as the other kids in the class,” she said.

“Knowing that this drug…could be just ripped away…the anguish we are living in is absolutely heartbreaking.”

A fortnightly 300mg dose of Brineura costs £522,722 for each patient every year, according to the National Institute for Health and Care Excellence (NICE).

An agreement on access to the drug between NHS England and manufacturer BioMarin is set to expire in May but NICE said “constructive” discussions over permanent access to the drug continued.

Wednesday’s rally in Westminster was backed by the Batten Disease Family Association and a supporter of the charity, the former England cricket captain Lord Botham, took part.

He said he hoped the government would keep the treatment going to allow children with the disease a “much better life”.

“It’s not curing, the research into it needs to be expanded and moved on because it is a brutal disease,” he told BBC Radio WM.