NEW HAVEN, Conn.--(BUSINESS WIRE)--Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) today announced that it has reached a national funding agreement with the National Institute for Health and Care Excellence (NICE) and the National Health Service (NHS) England based on a Managed Access Agreement (MAA), which provides access to Strensiq® (asfotase alfa) for patients in England with pediatric-onset hypophosphatasia (HPP), regardless of their current age. The funding agreement was announced today in a positive final evaluation determination (FED) issued by the NICE Highly Specialised Technologies (HST) Evaluation Committee to recommend Strensiq according to the MAA.
The MAA has been developed in collaboration between physician thought-leaders, patient groups, NHS England, and Alexion. The MAA ensures access to Strensiq for infants, children and adult patients with pediatric-onset HPP who experience the most disabling symptoms and are expected to benefit most from therapy.
“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,” said Lindsay Weaver, Chief Executive, Children Living with Inherited Metabolic Diseases (CLIMB). “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. We will be following the progress of the agreement, which involves the collection of robust data, to ensure continued access for patients.”
HPP is an ultra-rare metabolic disease characterized by defective bone formation that can lead to weakness and deformity of bones, fractures and other skeletal abnormalities, as well as complications such as profound muscle weakness, severe pain, seizures in perinatal/infantile forms of HPP, and respiratory failure potentially leading to premature death in infants.1-5
“We worked diligently and constructively with NICE, NHS England, advocates and physicians, and are extremely pleased that we were able to reach an agreement to make Strensiq available to patients with pediatric-onset HPP in England who are most in need of treatment,” said Ludwig Hantson, Chief Executive Officer of Alexion. "The decision to provide access to Strensiq is an important milestone for patients and their families."
Strensiq is approved in the European Union as a long-term enzyme replacement therapy in patients with pediatric-onset HPP. Strensiq is also approved in the United States for the treatment of patients with perinatal-, infantile- and juvenile-onset HPP, as well as in Japan and other countries. Alexion is currently progressing local funding processes for Strensiq in additional countries worldwide.
About Hypophosphatasia (HPP)
HPP is a genetic, chronic, progressive, and potentially life-threatening ultra-rare metabolic disease that can affect people of all ages. HPP is characterized by defective bone mineralization that can lead to weakness and deformity of bones, fractures and other skeletal abnormalities, as well as systemic complications such as profound muscle weakness, muscle, bone and joint pain, seizures in perinatal/infantile forms of HPP, and respiratory failure leading to premature death in infants. The signs, symptoms and severity of HPP can vary from patient to patient, and because of the progressive nature of the disease, new symptoms can appear at any age and symptoms can worsen over time, causing significant disability.1-5 HPP is traditionally classified by the age of the patient at the onset of symptoms of the disease, with perinatal-, infantile- and juvenile-onset HPP (also known as pediatric-onset HPP) defined by the onset of the first symptom prior to 18 years of age.1
HPP can have devastating consequences for patients at any stage of life.1 In a natural history study, infants who had their first symptom of HPP within the first 6 months of life had high mortality, with an overall mortality rate of 73 percent at 5 years.7 In these patients, mortality was primarily due to respiratory failure.1,7 In patients surviving and those with juvenile-onset HPP, long-term clinical sequelae include recurrent and non-healing fractures, profound muscle weakness, debilitating pain, and the requirement for ambulatory assistive devices such as wheelchairs, wheeled walkers, and canes.1,4
HPP is caused by mutations in the gene encoding an enzyme known as tissue non-specific alkaline phosphatase (TNSALP). This enzyme plays a critical role in the proper mineralization of bones.1,2
About Strensiq® (asfotase alfa)
Strensiq (asfotase alfa) is a highly innovative bone-targeted enzyme replacement therapy that treats the underlying cause of HPP by replacing the missing TNSALP enzyme. In clinical studies of patients with HPP who had their first symptom prior to the age of 18, treatment with Strensiq improved overall survival in infants, enhanced bone mineralization and improved height, weight, and mobility.6,8