The NHS in the UK has agreed to fund a drug for children with a rare degenerative disease, which leads to premature deaths.
The move will allow treatment of four children from Newcastle and Cheshire, who are suffering from Batten disease, an incurable illness that affects the nervous system, causing seizures, visual impairment and mobility loss.
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The disease usually starts in childhood, with an estimated 25 to 40 children living with the condition in England.
NHS England has agreed a deal with Biomarin, which has agreed a fairer price for the drug, cerliponase alfa.
Also known as Brineura, the drug is an enzyme replacement therapy administered directly into the brain through a surgical implant.
Cerliponase alfa has been demonstrated to extend the lives of youngsters who take it.
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By GlobalDataNHS England has said it will offer cerliponase alfa to sufferers who are not currently receiving treatment by Christmas.
NHS chief executive Simon Stevens said: “Over recent months, there have been a series of successful deals as NHS England works closely with the life sciences sector to make life-changing new drugs available for haemophilia, MS, rare cancers and other conditions.
“Coming after extended negotiation, the new deal reached today is a reminder that in order to succeed, companies must be flexible and realistic, because the NHS in England cannot and will not simply write blank cheques at taxpayers’ expense.”
The National Institute for Health and Care Excellence (NICE) will work with NHS England, BioMarin, clinicians and the Batten Disease Family Association on the details of the managed access agreement, which describes the patient eligibility criteria for access, as well as stopping rules and data collection arrangements.
