NEWS: Final NICE decision for Strensiq in the treatment of hypophosphatasia

July 2017 – After a 2 year process Climb is pleased that NICE has published draft guidance recommending the drug Strensiq (also known asfotase alfa) for people with paediatric-onset hypophosphatasia.


The funding agreement between NHS England and Alexion Pharma is based on a 5-year Managed Access Agreement which means that patients with pediatric onset hypophosphatasia (HPP) regardless of age who are most in need, will get access to this often life saving treatment.

This follows the recommendation of the drug asfotase alfa for children with perinatal- and infantile-onset HPP.

Climb has been the patient organisation consultee in the UK Government NICE process – representing people of all ages with HPP, from families with newborns through to adults living with the condition.  The Brittle Bone Society have also fully supported this process.

“We are extremely pleased to have been able to reach an agreement with NICE and NHS England to make Strensiq available to patients with pediatric-onset HPP in England who are most in need of treatment,” says Sara Trafford-Jones, Vice President and General Manager, UK and Ireland, Alexion Pharmaceuticals.  “Working with Climb and expert physicians has been a vital part of this process, ensuring that the needs of the patient has been truly reflected throughout the process and development of the Managed Access Agreement.”

Lindsay Weaver, Climb’s Chief Executive says “It is a success that patients with HPP in England who meet the criteria of the Managed Access Agreement will have access to Strensiq. This is the first therapy that specifically targets the underlying cause of hypophosphatasia and can save the lives of patients, usually babies who would otherwise not survive this devastating disease. It also dramatically changes the lives of those severely affected regardless of age. It has been a long process to get to this positive decision but we are relieved and delighted that this final agreement is the best outcome for this patient community.”

To continue our efforts we will be following the progress of the Managed Access Agreement, which involves the collection of information to support the evidence to continue access after the initial 5 year period.

The official draft recommendation by NICE can be read here.