Alexion Pharmaceuticals

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ABOUT ASFOTASE ALFA

(Formerly ENB-0040)

Asfotase alfa is a highly innovative human recombinant protein being developed by Alexion for the treatment of patients with HPP. HPP is an inborn error of metabolism arising from a genetic deficiency.¹ Patients with HPP cannot produce a form of the enzyme alkaline phosphatase known as tissue non-specific alkaline phosphatase (TNSALP), which is essential for regulating the phosphate levels in various metabolites that are critical for normal bone formation, and also for brain and muscle function.² The asfotase alfa drug candidate is a recombinant protein designed to address this genetic deficiency by targeting another form of alkaline phosphatase, known as functional alkaline phosphatase, directly to the affected patient tissues. In this way, asfotase alfa is designed to counter the genetically defective metabolic process and prevent or reverse the severe and life-threatening consequences of dysregulated calcium and phosphate metabolism in patients with HPP.

Data from two Phase 2 studies, one in infants and young children up to age 3 with severe HPP, and one in juveniles ages 5-12 with severe HPP, show marked improvement in bone development, muscle strength, and respiratory function with asfotase alfa treatment. In both studies, every patient showed an objective response to asfotase alfa treatment with an improvement in mineral metabolism.^1,3

Asfotase alfa was awarded orphan drug designation in the United States and European Union in 2008 and Fast Track status in the U.S. in 2009. If approved, asfotase alfa will be the first and only therapy to specifically address the underlying pathology of HPP through a targeted enzyme replacement therapy.

1. MP Whyte, et al. Hypophosphatasia in Children: Enzyme Replacement Therapy Using Bone-Targeted, Tissue-Nonspecific Alkaline Phosphatase. 2010 ASHG, Abst. 3070.
2. Mornet E. Review of Hypophosphatasia. Orphanet Journal of Rare Diseases. 2007:2:40 doi:10.1186/1750-1172-2-40. Available at: http://www.OJRD.com/content/2/1/40
3. CR Greenberg, et al. Life-Threatening Hypophosphatasia in Infants and Young Children: Results of Long-Term Treatment with ENB-0040, a Bone-Targeted, Enzyme Replacement-Therapy (ERT), and An Algorithm For Patient Management. 2010 ASHG, Abst. 13.