Rare Daily Staff

Alexion Pharmaceuticals has submitted an application to the U.S. Food and Drug Administration for the approval of ALXN1210, the company’s experimental long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria, a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder.

In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in the destruction of red blood cells, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath. The most devastating consequence of this is the formation of blood clots, which can damage vital organs and cause premature death.

ALXN1210 works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system that, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders like PNH, atypical hemolytic uremic syndrome, and anti-acetylcholine receptor antibody-positive myasthenia gravis.

Alexion is using a rare disease priority review voucher, which provides for an expedited eight-month review by the FDA instead of the standard 12-month review.

“This first regulatory submission is an important step toward our goal of establishing ALXN1210 as the new standard of care for patients with PNH, building on 10 years of proven efficacy and safety with Soliris, and 25 years of leadership in complement biology,” said John Orloff, executive vice president and head of research & development at Alexion.

The application is supported by comprehensive data from two phase 3 clinical trials in more than 440 patients, which included patients who had never received a complement inhibitor, and patients who were stable on Soliris and switched to ALXN1210.

Alexion is also preparing submissions for the approval of ALXN1210 as a treatment for patients with PNH in the European Union by mid-year and in Japan in the second half of the year. ALXN1210 has received Orphan Drug Designation for the treatment of patients with PNH in the U.S. and EU.

ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and EU, and for the subcutaneous treatment of patients with aHUS in the U.S.

June 20, 2018
John Orloff, executive vice president and head of research & development at Alexion