Articles from Alexion

VOYDEYA™ (danicopan) has been approved in the US as add-on therapy to ravulizumab or eculizumab for the treatment of extravascular hemolysis (EVH) in adults with paroxysmal nocturnal hemoglobinuria (PNH).1 VOYDEYA is a first-in-class, oral, Factor D inhibitor developed as an add-on to standard-of-care ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) to address the needs of the approximately 10-20% of patients with PNH who experience clinically significant EVH while treated with a C5 inhibitor.2,3

ULTOMIRIS® (ravulizumab-cwvz) has been approved in the United States (US) as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).1

Positive results from the 24-week and long-term extension (LTE) period of the pivotal ALPHA Phase III trial showed danicopan as add-on to standard of care C5 inhibitor therapy ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) continued to demonstrate clinical benefit for patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH).1

Detailed positive results from the Phase III CHAMPION-NMOSD trial showed that ULTOMIRIS® (ravulizumab-cwvz) significantly reduced relapse risk in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD), compared to the external placebo arm from the pivotal SOLIRIS® PREVENT clinical trial. Data were presented today at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress.

Alexion, AstraZeneca Rare Disease, will present new data showing significant advances for the treatment of anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD) at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress, October 26 to 28, 2022.

Alexion, AstraZeneca Rare Disease, today announced that it has entered a definitive agreement to acquire Lexington, Mass.-based LogicBio® Therapeutics, Inc. (NASDAQLOGC), a pioneering genomic medicine company. The proposed acquisition aims to rapidly accelerate Alexion’s growth in genomic medicines through LogicBio’s unique technology, experienced rare disease R&D team, and expertise in pre-clinical development.

A prespecified interim analysis of the ALPHA Phase III trial evaluating danicopan (ALXN2040), an investigational, oral factor D inhibitor, as an add-on to C5 inhibitor therapy ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS® (eculizumab) showed positive high-level results in patients with paroxysmal nocturnal hemoglobinuria (PNH) who experience clinically significant extravascular hemolysis (EVH).

Detailed results from the positive FoCus Phase III trial in Wilson disease showed that ALXN1840, an investigational once-daily, oral medicine, met its primary endpoint demonstrating three-times greater copper mobilization from tissues compared to the standard of care (SoC) arm (Least Square Mean Difference [LSM Diff] 2.18 µmol/L; p< 0.0001), including in patients who had been treated previously for an average of 10 years.1