FDA Accepts Alexion’s Experimental PNH Treatment for Priority Review

Rare Daily Staff

The U.S. Food and Drug Administration has accepted for review Alexion Pharmaceuticals’ application for 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.

The FDA set a target date to act on the application of February 18, 2019. It reflects an expedited eight-month review instead of the standard 12-month review following Alexion’s use of a rare disease priority review voucher.

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 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.

The application is supported by comprehensive data from two phase 3 clinical trials.

“We are working with the FDA to facilitate a smooth review,” said John Orloff, executive vice president and head of research and development at Alexion. “Building on comprehensive results from the largest-ever phase 3 development program in PNH, 11 years of proven efficacy and safety with Soliris, and 25 years of leadership in complement biology, we are on track with our efforts to establish ALXN1210 as the new standard of care for patients with PNH.”

If approved, ALXN1210 would be the first and only long-acting complement inhibitor for patients with PNH, providing immediate and complete inhibition of the C5 complement protein that is sustained over an eight-week dosing interval.

In addition to the submission in the United States on June 18 and the submission in the European Union on June 28, Alexion is preparing a submission for a New Drug Application for ALXN1210 as a treatment for patients with PNH in Japan in the second half of the year. The European Medicines Agency has accepted and is reviewing the submission for the EU. ALXN1210 has received Orphan Drug Designation in the United States and European Union for the treatment of patients with PNH.

August 20, 2018
Photo: John Orloff, executive vice president and head of research and Development at Alexion