Alexion Pharma International Sàrl, a subsidiary of Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN), today announced that the U.S. Food and Drug Administration (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. HPP is an inherited, life-threatening, ultra-rare metabolic disorder that leads to progressive damage to multiple vital organs, including destruction and deformity of bones.
“Asfotase alfa is a highly innovative therapeutic candidate with the potential to transform the lives of patients with HPP who currently have no treatment options and often receive only palliative care for this life-threatening disease.”
The FDA also confirmed that adult-onset HPP is "a serious and life threatening disease or condition" and that Breakthrough Therapy designation could be obtained for this aspect of the disease with additional clinical information.
According to the FDA, a Breakthrough Therapy designation is designed to expedite the development of a drug to treat a serious or life-threatening disease when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. The Breakthrough Therapy designation is part of the FDA Safety and Innovation Act (FDASIA) of 2012.
"The FDA's Breakthrough Therapy designation for perinatal-, infantile- and juvenile-onset HPP recognizes the severe, debilitating and life-threatening nature of the disease, the clear unmet medical need of patients, and the clinical evidence collected to date on asfotase alfa," said Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D at Alexion. "Asfotase alfa is a highly innovative therapeutic candidate with the potential to transform the lives of patients with HPP who currently have no treatment options and often receive only palliative care for this life-threatening disease."
Alexion looks forward to working closely with the FDA and obtaining FDA guidance on the subsequent development of asfotase alfa for the treatment of HPP, including obtaining advice on generating evidence needed to support approval of the drug in an efficient manner. Clinical studies of asfotase alfa are ongoing for patients with HPP whose first signs or symptoms occurred prior to 18 years of age, including perinatal-, infantile- and juvenile-onset forms of HPP.
SOURCE Alexion Pharmaceuticals, Inc.