NICE recommends Strensiq (asfotase alfa)to treat perinatal and infantile-onset hypophosphatasia but not for people with hypophosphatasia that appears later in childhood or in adults.Alexion Pharma

NICE (The National Institute for Health and Care Excellence) has published draft guidance recommending the drug Strensiq (asfotase alfa) for children with perinatal- and infantile-onset hypophosphatasia � a very rare inherited condition affecting between one and seven babies each year in England. The committee concluded that asfotase alfa improved the probability of survival in perinatal- and infantile-onset hypophosphatasia compared with best supportive care.

The draft guidance recommends asfotase alfa for children with the condition as part of a managed access agreement and when the company provides the drug with the proposed cost cap which will limit the cost the NHS will have to pay per patient.The five-year period of the agreement will allow more information to be gathered on how long treatment should continue, and when treatment could be stopped or the dose reduced before the guidance is reviewed and a further decision made on whether funding should be continued.

The draft guidance does not recommend the drug for people with hypophosphatasia that appears later in childhood or in adults. This was because the committee felt that the treatment need for these people varied, and the criteria for starting and stopping treatment with asfotase alfa in the managed access agreement did not identify those who would benefit the most within this group.