Drugs to Watch: Regulatory Focus – Tarpeyo/Kinpeygo

Orphan drug received accelerated approval based on a surrogate study endpoint

Tarpeyo®/Kinpeygo® (developed under the project name Nefecon) is the first FDA-approved drug to decrease urine protein in immunoglobulin A nephropathy (IgAN), a rare kidney disease. Previously designated an orphan drug, it was granted accelerated approval in 2021 based on a surrogate endpoint (the treatment effect on proteinuria) rather than demonstration of clinical benefit via endpoints such as overall response rate (ORR) or duration of response (DOR). This resulted in a limited initial indication; however, Tarpeyo was later given an expanded indication supported by follow-up clinical trial data, dramatically broadening its treatable population. 

This report explores:

Disease and competitive landscape
Regulatory strategy (e.g., which pathways to pursue in key markets)
Key areas of regulatory focus, such as safety concerns, pharmacovigilance, use in specific populations and effectiveness
Outstanding safety and efficacy concerns