AstraZeneca's Add On Treatment With Standard Care Improves Hemoglobin Levels In Rare Blood Disorder - Ast

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2023-06-09 12:44:45  • 3 mins

AstraZeneca PlcAZNreleaseddata from the Phase 3 ALPHA trial of danicopan as an add-on to the standard of care for paroxysmal nocturnal hemoglobinuria (PNH).

The data showed that danicopan as an add-on to the standard of care C5 inhibitor therapy(ravulizumab) or(eculizumab) demonstrated a statistically significant and clinically meaningful increase in hemoglobin levels and maintained disease control in PNH patients, compared to placebo plus established C5 inhibitor therapy

PNH is a rare and severe blood disorder characterized by the destruction of red blood cells within blood vessels, known as intravascular hemolysis (IVH), and white blood cell and platelet activation that can cause thrombosis (blood clots).

In patients managing PNH withor, add-on treatment with danicopan was superior to placebo plusorbased on the change in hemoglobin from baseline to week 12.

Further, significant improvements in hemoglobin were observed with danicopan by week two and maintained through week 12.

All key secondary endpoints also met statistical superiority in favor of danicopan plusorcompared to placebo plus C5 inhibition.

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