Submission Updates
A momentous milestone for people living with Hypophosphatasia We were delighted to share in February that following a second committee meeting, NICE released final draft guidance to recommend the use of Asfotase alfa (Strensiq) for the treatment of all patients with perinatal and infantile forms of paediatric-onset hypophosphatasia (HPP) and additional recommendations for those with juvenile forms who meet the entry requirements set by the Managed Access Agreement (MAA).
So, what is Strensiq?
Strensiq is a drug that is life-saving in the youngest and life-changing in older children, young people, and adults with paediatric-forms of HPP. We share in the emotion, the relief, and the future hope that this guidance brings all of those living with HPP their families, and all those involved in their care.
Our Individual Support Manager, Helen Morris, has steered this work from within MSUK with great attention, care, and emotion. We have been privileged to represent the HPP community throughout this process and to work alongside those affected by HPP, their parents and carers, Soft Bones in the UK and US, and metabolic bone consultants and other specialities. For the HPP community this process has been long and there have been many uncertainties along the way. We have worked with NICE and NHS England to unpick each stage and what it means for those receiving Strensiq and we are absolutely delighted that this new guidance relieves a great deal of anxiety and concern for many.
A key message here is that in the case of rare disorders, no matter how little you feel you can share, your voice really does matter! We are celebrating this huge success as a result of the time, effort and dedication of those living with HPP and their families and carers. Throughout this process they have completed questionnaires not just as part of the MAA but from MSUK too. These, and the interviews, calls, and emails sharing vital insights and experiences of life with HPP have been what has got this over the line and helped us to ensure decision makers understand the true impact of HPP and the differences Strensiq has made.
