Bardoxolone Gets Orphan Drug Status for Autosomal Dominant Polycystic Kidney Disease

The Food and Drug Administration (FDA) has granted bardoxolone methyl (bardoxolone; Reata Pharmaceuticals) Orphan Drug designation for the treatment of autosomal dominant polycystic kidney disease (ADPKD).

Bardoxolone is an activator of nuclear 1 factor erythroid-derived 2–related factor 2 (Nrf2), a transcription factor that induces molecular pathways that promote restoration of mitochondrial function, reduction of oxidative stress, and inhibition of pro-inflammatory signaling.

Earlier this year, Reata shared positive results from the phase 2 PHOENIX study which included 31 patients with ADPKD. Following 12 weeks of treatment with bardoxolone, a mean increase from baseline in estimated glomerular filtration rate (eGFR) of 9.3mL/min/1.73m² (P<.0001) was observed in this patient population; the other cohorts included patients with IgA nephropathy, type 1 diabetic chronic kidney disease, and focal segmental glomerulosclerosis.

A phase 3, placebo-controlled study (FALCON) is currently recruiting patients with ADPKD with eGFR between 30-90mL/min/1.73m² to investigate the safety, tolerability and efficacy of bardoxolone; the primary efficacy endpoint of the study is the change from baseline in eGFR compared with placebo after 48 weeks of treatment followed by a 4-week drug withdrawal period.

“We observed significant improvements in kidney function in the ADPKD cohort of the phase 2 PHOENIX study, and historically we have observed strong correlations between changes in eGFR after 12 weeks of bardoxolone treatment and 1-year retained eGFR benefit in other forms of chronic kidney disease,” said Warren Huff, Reata’s Chief Executive Officer and President. “We are hopeful that bardoxolone may serve as a meaningful new treatment option for patients with ADPKD.”

For more information visit Reatapharma.com.