NICE has published further draft guidance as part of its highly specialised technologies programme recommending asfotase alfa (Strensiq, Alexion Pharma UK) for children with hypophosphatasia. A rare, and often fatal genetic condition.

The drug is the first treatment to target the underlying cause of perinatal- and infantile-onset hypophosphatasia, Asfotase alfa provides long-term enzyme replacement therapy to enable minerals such as calcium and phosphorous to be deposited in developing bones and teeth again, and helps minimised complications of dysregulated bone mineral metabolism.

In England it is estimated between one and seven people are diagnosed with perinatal- and infantile-onset hypophosphatasia each year.

Hypophosphatasia is an inherited disorder that disrupts minerals such as calcium and phosphorus being deposited in developing bones. Infants with perinatal- and infantile-onset hypophosphatasia have a high mortality rate, with up to 50%-100% dying within the first year of life, primarily due to respiratory failure. Symptoms and severity can greatly vary for children who develop it later in childhood.

Asfotase alfa has been described as a ‘step-change’ in the management of the condition, offering “salvation” for babies and young children that could permit a good quality of life, but without which many would not survive.

The drug’s list price is £58.80 per mg (excluding VAT), meaning that it would cost £165,000 per year for babies and as much as £1.5 million per year for adults and young people. The draft guidance also states that the company should provide further opportunities to reduce the short-term cost of asfotase alfa to the NHS.

Professor Carole Longson MBE, NICE health technology evaluation centre director, said, “Paediatric-onset hypophosphatasia is a serious, life-threatening and debilitating condition that has severe effects on the lives of people with the condition, as well as their families and carers. Based on the evidence presented by the company, as well as the testimony of clinical experts and patient representatives, the committee concluded that asfotase alfa improved the probability of survival in perinatal- and infantile-onset hypophosphatasia compared with best supportive care.

“The committee also accepted that asfotase alfa was likely to be clinically effective across a range of outcomes, such as reducing the need for respiratory support and the severity of rickets for people with perinatal- and infantile-onset hypophosphatasia.”

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