UK’s National Institute for Health and Care Excellence (NICE) has recommended the use of Alexion Pharma’s Strensiq (asfotase alfa) drug for the treatment of people with paediatric-onset hypophosphatasia.

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The organisation has published draft guidance recommending the use of the drug to treat the rare inherited life-threatening disease claimed to affect between one and seven babies each year in England.

Hypophosphatasia disrupts the process in which minerals such as calcium and phosphorus are deposited in developing bones in human body.

Asfotase alfa offers long-term enzyme replacement therapy to restore normal development of bones and teeth.

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The NICE draft guidance previously recommended the Strensiq treatment for the babies who were affected with the most severe form of the disease.

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Alexion has entered a new agreement with National Health Service (NHS) England on price and access to the treatment, meaning NICE can currently recommend it for a broader group of people eligible for treatment on the NHS.

"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."

The recent deal includes a five-year managed access agreement, which reduces the cost of the drug to the NHS patients and allows people with the highest unmet need to be identified.

Alexion Pharma’s Strensiq is the first therapy that particularly targets the underlying cause of hypophosphatasia.

NICE Centre for Health Technology Evaluation director Carole Longson said: “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.”

Under the deal, Strensiq will be made available on the NHS to eligible children and adults for an initial period of five years, during which more data will be gathered on how long the treatment should be continued.

Image: NICE broadens the use of Alexion’s paediatric-onset hypophosphatasia treatment on NHS patients. Photo: courtesy of NICE.