Drugs to Watch – Regulatory in Focus – Casgevy and Lyfgenia

Groundbreaking gene therapies with fast-track approval pose unique challenges for clinical trials

Casgevy™ and Lyfgenia™ are the first FDA-approved cell-based gene therapies for the treatment of sickle cell disease (SCD). Approved under expedited programs for serious conditions, they represent groundbreaking gene therapies with unique considerations for regulators—and a unique approach to study design for ethical reasons. Despite impressive study results, the FDA expressed safety concerns, indicating the need for additional studies and postmarketing surveillance.

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