Alexion Pharmaceuticals said that it has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA), seeking approval for its C5 complement inhibitor ALXN1210 for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH).
The submission uses a rare disease priority review voucher, which designates the BLA for an expedited eight-month review by the FDA instead of the standard 12-month review.
Alexion executive vice president and research & development head John Orloff said: “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.
“We look forward to working with the FDA to facilitate a rapid review.”
The application is supported by comprehensive data from two rigorous Phase 3 clinical trials in the largest population of patients with PNH ever studied: more than 440 patients, which included patients who had never received a complement inhibitor, and patients who were stable on Soliris (eculizumab) and switched to ALXN1210.
Weight-optimized treatment with ALXN1210 every eight weeks demonstrated non-inferiority to treatment every two weeks with Soliris on all primary endpoints and key secondary endpoints in both studies.
The numeric results for all these endpoints, including breakthrough hemolysis (one of the key secondary endpoints), favored ALXN1210 in both studies and are consistent with the immediate and complete C5 inhibition observed by the end of the first infusion of ALXN1210 and sustained throughout the entire 26-week treatment period.
There were no notable differences in the safety profiles for ALXN1210 and Soliris. Topline data of these Phase 3 studies were disclosed in press releases on March 15, 2018 and April 26, 2018, respectively.
In addition to the BLA in the U.S., Alexion is preparing submissions for the approval of ALXN1210 as a treatment for patients with PNH in the European Union (EU) by mid-year and in Japan in the second half of the year.
ALXN1210 has received Orphan Drug Designation (ODD) for the treatment of patients with PNH in the U.S. and EU.
Source: Company Press Release