FDA Grants Accelerated Approval to Kresladi for Severe LAD-I
On March 27, the U.S. FDA approved Rocket Pharmaceuticals’ gene therapy Kresladi for severe Leukocyte Adhesion Deficiency-I (LAD-I).
Kresladi is the first approved gene therapy for LAD-I. The decision marks an important step forward for gene therapy in ultra-rare diseases, where both clinical value and execution capability remain critical.
Key points
- First approved gene therapy for LAD-I
- Approved under the accelerated approval pathway
- 100% overall survival at 12 months in the Phase 1/2 study
- Reduced severe infections and improved wound healing were observed
Industry implications
This approval is significant in showing that gene therapy can reach regulatory approval even in ultra-rare diseases. Going forward, success will depend not only on scientific innovation, but also on execution across CMC, GMP manufacturing, quality management, and regulatory strategy.
Greenstaff Life Sciences extend our congratulations to Rocket Pharmaceuticals and to everyone involved in bringing this therapy forward.