The FDA has approved Rocket Pharma's gene therapy Kresladi for treatment of a rare immune disorder. The approval comes after testing in a small patient population of nine individuals. The therapy showed positive results in clinical trials despite the limited sample size.
Gene therapies for rare diseases often face the challenge of small patient populations, making traditional large-scale clinical trials difficult to conduct. The FDA has established pathways for approving treatments for rare conditions based on smaller datasets when the medical need is significant and safety profiles are acceptable.