NICE has published draft guidance recommending the drug asfotase alfa for children with perinatal- and infantile-onset hypophosphatasia (HPP) – a very rare inherited condition affecting between one and seven babies each year in England.
The full draft guidance can be accessed here. In summary:
- Asfotase alfa is recommended as an option for treating the bone manifestations of hypophosphatasia, only: in people with perinatal- and infantile-onset disease and for the duration of, and within the conditions set out in, the proposed managed access agreement.
- Asfotase alfa is not recommended for treating the bone manifestations of hypophosphatasia in children with juvenile-onset disease.
- Asfotase alfa is not recommended for treating the bone manifestations of hypophosphatasia in adults with juvenile-onset disease.
Climb has been patient organisation consultee in the UK Government NICE process – assessing the drug afostase alfa for people with HPP. The Brittle Bone Society have also fully supported this process.
Climb has represented patients of ALL ages with HPP from families with newborns through to adults living with the condition. It is our aim to continue to do this in view of the latest outcome. Climb will continue to work in partnership with all stakeholders to get the best possible results for patients and their families.
Lindsay Weaver, Climb’s Executive Director says:
“Whilst we are extremely pleased that asfotase alpha is recommended for children with perinatal and infantile-onset hypophosphatasia, we are disappointed that is not recommended for those where HPP appears later in childhood or in adults. These forms of the condition can have a severe impact on the lives of those affected.
As official consultee representing patients with HPP of all ages, We do feel that the criteria for starting and stopping treatment with asfotase alfa in the managed access agreement did identify those who would benefit the most within this group and will provide a thorough response to offer further assurance to NICE on this.”
The deadline for responding to the recommendation closed on 13th October 2016 and we await the final decision. The official response of Climb and the Brittle Bone Society can be read here: OFFICIAL RESPONSE – HPP