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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
