The FDA has expanded approval of the CRISPR-based gene therapy Casgevy to include children as young as 5 with severe sickle cell disease, offering a potential one-time cure for the painful genetic condition.
Clinical Results
In pediatric trials, 94% of treated children were free of vaso-occlusive crises for at least 18 months post-treatment. Quality of life scores improved by 80% on average.
Access Challenges
The treatment costs $2.2 million per patient. Insurers are negotiating outcomes-based payment models where full payment is contingent on long-term efficacy.
- Approved for children ages 5+
- 94% free of pain crises at 18 months
- One-time treatment, potential cure
- Cost: $2.2M per patient
