Adjunct Danicopan for PNH with EVH Maintains Superiority Versus Placebo in ALPHA

Danicopan as an add-on to standard-of-care complement component 5 (C5) inhibitor therapy among patients with paroxysmal nocturnal hemoglobinuria (PNH) with clinically significant extravascular hemolysis (EVH) continued to show treatment benefits during the 24-week and long-term extension outcomes in the phase III ALPHA trial.

“Expanding on positive 12-week results, the findings demonstrate sustained improvements in hemoglobin levels for up to 48 weeks, while also maintaining disease control, as measured by lactate dehydrogenase levels,” said ALPHA investigator Austin Kulasekararaj, MD, at King’s College Hospital in London.

Continued Evidence of Benefit with Danicopan Treatment in PNH

The open-label and long-term extension data showed that significant improvements in hemoglobin reported in the prespecified interim analysis at week 12 were maintained through both the 24-week and 48-week analyses.

Additional endpoints with improvements sustained from week 12 to 24 included change in baseline from hemoglobin, absolute reticulocyte count, lactate dehydrogenase levels, percentage of patients with hemoglobin increases of ≥2 g/dL without transfusion, and percentage of patients with transfusion avoidance.

“These new data further demonstrate the potential of danicopan as add-on to [ravulizumab] or [eculizumab] to address the needs of the small subset of patients with PNH who experience clinically significant EVH,” Dr. Kulasekararaj and colleagues concluded.

Related: Add-on Factor D Inhibitor Improves Hemoglobin Levels in PNH with Hemolysis

Reference

Kulasekararaj A, Griffin, Piatek CI, et al. Danicopan as add-on therapy to ravulizumab or eculizumab versus placebo in patients with paroxysmal nocturnal hemoglobinuria and clinically significant extravascular hemolysis: phase 3 long-term data. Blood. doi: https://doi.org/10.1182/blood-2023-189863