Question to the Department of Health and Social Care:

To ask Her Majesty’s Government, in the light of the draft recommendation from NICE not to recommend the drug Strensiq for the treatment of hypophosphatasia for critically ill infants who are otherwise likely to die, what other treatment options are available.

The National Institute for Health and Care Excellence (NICE) is currently evaluating Strensiq (asfotase alfa) for the treatment of paediatric-onset hypophosphatasia under its highly specialised technologies programme.

NICE’s draft guidance, published in December 2015 for consultation, did not recommended asfotase alfa. However this is not NICE’s final guidance to the National Health Service.

NICE’s draft guidance states that there are currently no other treatments available that specifically prevent or delay the progression of hypophosphatasia.