Pediatric-onset hypophosphatasia (HPP), an extremely rare metabolic disease affecting bone structure and strength, came into sharper focus recently when Alexion Pharmaceuticals won funding for its Strensiq medication from two sources via a managed access agreement.
In the agreement with Connecticut-based Alexion, the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England pledged to ensure accessibility of Strensiq (asfotase alfa) for patients of all ages demonstrating need for symptom alleviation.
“It is a success that patients with HPP in England who meet the criteria of the Managed Access Agreement will have access to Strensiq, which is the only treatment for this severely debilitating and often life-threatening disease,” Lindsay Weaver, chief executive of Children Living with Inherited Metabolic Diseases (CLIMB), said.
Strensiq has already won approval in the European Union for individuals with pediatric-onset HPP as an enzyme replacement therapy. In the U.S., it is also authorized for treatment in those with perinatal-, infantile- and juvenile-onset HPP, according to the company, which is pursuing additional funding for Strensiq in nations worldwide.
“We are relieved that NICE, NHS England and Alexion have reached an agreement that benefits patients with pediatric-onset HPP most in need of treatment,” Weaver said. “We will be following the progress of the agreement, which involves the collection of robust data, to ensure continued access for patients.”
Alexion CEO Ludwig Hantson also expressed satisfaction at the collaborative arrangement, saying, "The decision to provide access to Strensiq is an important milestone for patients and their families."
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