Asfotase alfa is an investigational, first-in-class targeted enzyme replacement therapy for the treatment of hypophosphatasia (HPP), a genetic, chronic and progressive ultra-rare metabolic disease that can lead to destruction and deformity of bones, profound muscle weakness, seizures, respiratory failure and premature death.
In this exclusive interview with Rare Disease Report, Martin Mackay, PHD, global head of research and development at Alexion Pharmaceuticals talks about the results of their clinical studies with asfotase alfa to treat HPP.
1. Whyte MP, Rockman-Greenberg C, Hofmann C, et al. Improved survival with asfotase alfa treatment in pediatric patients with hypophosphatasia at high risk of death. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting, Houston, September 14, 2014. Abstract 1097.
2. Whyte MP, Simmons JH, Lutz RE. Enzyme-replacement therapy in life-threatening hypophosphatasia: The 3-year experience with asfotase alfa. Poster presented at the American Society for Bone and Mineral Research (ASBMR) 2014 Annual Meeting, Houston, September 12, 2014. Abstract FR0435.