Biogen reports trial results for kidney disease drug in adolescents

Biogen Inc. has shared positive results from a study examining the effects of its drug EMPAVELI on adolescents with kidney diseases. The Phase 3 VALIANT study focused on 55 patients aged 12 to 17 years with C3 glomerulopathy or primary immune complex membranoproliferative glomerulonephritis. By Week 26, those taking EMPAVELI experienced a 75% reduction in proteinuria compared to those receiving a placebo.

Moreover, 71% of the EMPAVELI group saw a 50% or greater decrease in proteinuria, versus just 4% of the placebo group. Notably, 57% of adolescents on EMPAVELI maintained stable kidney function alongside significant proteinuria reduction, as opposed to just 4% of the placebo group. These improvements were evident as early as Week 4 and persisted through Week 26 of the trial.

EMPAVELI was administered twice weekly to participants for 26 weeks, followed by an additional 26 weeks of open-label treatment. The FDA recently expanded EMPAVELI's approval to include adolescents aged 12 and older, citing its efficacy in reducing both proteinuria and kidney function decline. The drug demonstrated a favorable safety profile in adolescents, aligning with the overall VALIANT study's findings.

C3G and primary IC-MPGN are rare, chronic conditions where about 20% of children progress to kidney failure within a decade of diagnosis, despite standard treatments.