Alexion announced that the FDA has 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.
Asfotase alfa is an investigational, first-in-class targeted enzyme replacement therapy designed to address the underlying cause of HPP by normalizing the genetically defective metabolic process, and preventing or reversing the severe and life-threatening complications of life-long dysregulated mineral metabolism.
Alexion will work closely with the FDA to obtain guidance on the development of asfotase alfa for the treatment of HPP and collecting evidence needed to support approval of the drug in an efficient manner. Clinical studies of asfotase alfa are ongoing for patients with HPP.
For more information call (203) 272 – ALXN or visit www.alexionpharma.com.