PNH is a debilitating, ultra-rare, life-threatening blood disorder in which uncontrolled activation of complement, a component of the immune system, results in hemolysis (destruction of a patient’s red blood cells).
Alexion executive vice president and global head Martin Mackay, Ph.D said: “Alexion is committed to achieving the highest levels of innovation to address the needs of patients suffering from PNH, a devastating ultra-rare disorder.
“We are pleased that the FDA has recognized the potential for ALXN1210 to offer a significant therapeutic advantage for patients with PNH. Data from our ongoing clinical studies have shown rapid, complete, and sustained complement inhibition in treated patients, and we look forward to continuing to evaluate this highly innovative molecule in our Phase 3 trial of ALXN1210 administered every eight weeks.”
Alexion is currently enrolling patients in Phase 3 trials of ALXN1210 in patients with PNH as well as in patients with atypical hemolytic uremic syndrome (aHUS), another ultra-rare and life-threatening disease caused by chronic uncontrolled complement activation.
In June 2016, ALXN1210 was granted ODD by the European Commission for the treatment of patients with PNH. ALXN1210 is protected by a composition of matter patent in the U.S. and Europe through 2035. ALXN1210 is not approved in any country.
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.
ODD provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.