Dr. Martin Mackay Appears on "BioCentury This Week" to Discuss Breakthrough Therapy Designation for Asfotase Alfa for the Treatment of Patients with Hypophosphatasia
Alexion currently has nine lead development programs underway with its five highly innovative therapeutic candidates, including asfotase alfa, a first-in-class targeted enzyme replacement therapy in Phase 2 clinical trials for Hypophosphatasia (HPP). HPP is an inherited and life-threatening ultra-rare metabolic disorder that leads to progressive damage to multiple vital organs, including destruction and deformity of bones.
In May 2013 the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to asfotase alfa for the treatment of patients with hypophosphatasia (HPP) whose first signs or symptoms occurred prior to 18 years of age, including perinatal-, infantile-, and juvenile-onset forms of the disease. Dr. Martin Mackay appeared on BioCentury This Week to discuss Breakthrough Therapy designation for asfotase alfa. Watch Video