A drug which would be a lifeline for children with an extremely rare inherited condition has been approved by NICE in an unprecedented step forward.
Paediatric-onset hypophosphatasia disrupts the process of calcium and phosphorus being deposited into developing bones, stunting development of bones and teeth. It affects between one and seven babies in England each year.
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Its high mortality rate sees up to 100% of babies affected dying in the first year of life. Asfotase alfa, a drug which provides a long term enzyme replacement therapy to restore normal development, has now been approved for use in children and adults.
Director for health technology evaluation at NICE Professor Carole Longson said: ‘Asfotase alfa is an important development in the treatment of this devastating condition that has been shown to save lives and prevent or delay its progression.
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‘The new deal, which includes a managed access agreement, between the company and NHS England means that people with the greatest clinical need for treatment can be identified and the costs and risks to the NHS have been reduced.
‘We are very pleased therefore to be able to recommend it as an option for treating paediatric-onset hypophosphatasia.’
NICE was initially reluctant to permit use of the drug as its benefits were uncertain, but has now struck a deal with the manufacturer which will allow those with the highest unmet need access to treatment.
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The agreement means asfotase alfa will be made available on the NHS to eligible children and adults for 5 years initially. During this time more information will be gathered on how long treatment should continue, and when treatment could be stopped or the dose reduced.