The U.S. Food and Drug Administration (FDA) on Thursday granted accelerated approval to AstraZeneca Plc’s (NYSE:AZN) Ultomiris (ravulizumab) to lower proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of disease progression.
Accelerated FDA Clearance Based On Proteinuria Reduction
The agency cleared the medicine under its accelerated pathway based specifically on reductions in proteinuria. Regulators and researchers have not yet established whether Ultomiris slows kidney function decline in IgAN patients.
Therefore, continued marketing approval for this indication remains contingent upon the verification and description of clinical benefit in the ongoing Phase 3 I CAN clinical trial.
Interim Phase 3 Trial Shows Significant Efficacy
The regulatory clearance followed a Priority Review driven by positive interim findings from the I CAN study.
Ultomiris demonstrated a statistically significant placebo-adjusted proteinuria reduction of 43.4% at 34 weeks. Treated patients achieved a 46.6% drop in 24-hour urine protein creatinine ratio, compared to a 5.6% decline among placebo recipients.
Proteinuria reduction occurred rapidly, appearing as early as week 10 with a 36.7% drop versus 8.5% for placebo, and remained sustained through 34 weeks. These clinical outcomes proved consistent across patient subgroups, regardless of demographic background or baseline disease severity.
Qualified adult patients receive the intravenous infusion every eight weeks following a weight-based loading dose.
Addressing High-Risk Kidney Disease
IgAN is a rare, inflammatory kidney disorder impacting more than 200,000 people in the U.S.
The illness develops when abnormal IgA proteins form immune deposits in the kidneys, activating the complement system and causing terminal complement-driven inflammation. This process damages glomeruli cells responsible for filtering and cleaning blood.
Approximately half of patients presenting with elevated urinary protein or reduced kidney performance progress to end-stage kidney disease within 10 years of diagnosis.
The Phase 3 I CAN study remains ongoing, evaluating changes in estimated glomerular filtration rate at week 106 to support traditional FDA approval.
AZN Price Action: AstraZeneca shares were up 2.49% at $172.65 at the time of publication on Thursday, according to Benzinga Pro data.
Photo by Piotr Swat via Shutterstock
Recent Comments