1 Recommendations

1.1 Asfotase alfa is recommended as an option for treating paediatric-onset hypophosphatasia only:

  • for people who meet the criteria for treatment within the managed access arrangement (see section 4.18), and

  • for the duration of this arrangement and in line with the other conditions it specifies, and

  • when the company provides asfotase alfa with the confidential commercial terms agreed with NHS England.

1.2 These recommendations are not intended to affect treatment with asfotase alfa that was started in the NHS before this guidance was published. People having treatment outside these recommendations may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. For children and young people, this decision should be made jointly by the clinician and the child or young person or the child or young person's parents or carers.

  • National Institute for Health and Care Excellence (NICE)