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.
“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.