FDA Rejects Rocket Pharmaceuticals’ Gene Therapy for Ultra-Rare Immune Disease Over Manufacturing Concerns

1. FDA Rejection: The FDA has rejected Rocket Pharmaceuticals' gene therapy, Kresladi, for the treatment of leukocyte adhesion deficiency-I (LAD-I), a rare and severe pediatric disorder.
2. Manufacturing Concerns: The FDA requested additional information on the therapy's Chemistry Manufacturing and Controls (CMC) to ensure consistency in safety, purity, potency, and performance across all batches.
3. Limited Additional Information: The FDA asked for "limited additional" manufacturing information, indicating that the company needs to provide more data to address the agency's concerns.
4. Positive Clinical Results: The gene therapy has shown positive results in a phase I/II study, demonstrating 100% overall survival for LAD-I patients at 12 months post-treatment.
5. Potential Alternative: If approved, Kresladi could offer a less risky and more accessible treatment option for LAD-I patients compared to the current standard treatment, allogeneic bone marrow transplant.
6. Company Response: Rocket Pharmaceuticals remains committed to addressing the FDA's concerns and is planning to initiate clinical studies for a new gene therapy candidate aimed at treating dilated cardiomyopathy later this year.