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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
