The U.S. Food and Drug Administration has granted accelerated approval for Kresladi (marnetegragene autotemcel), marking it as the first gene therapy approved for treating severe Leukocyte Adhesion Deficiency Type I (LAD-I). This approval is granted to Rocket Pharmaceuticals, Inc.
Job Overview
Kresladi is indicated for pediatric patients with severe Leukocyte Adhesion Deficiency I (LAD-I) due to biallelic variants in ITGB2, who do not have an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant. As a condition of the accelerated approval, Rocket Pharmaceuticals, Inc. is required to conduct post-approval studies to verify and describe the clinical benefit of Kresladi.
Eligibility Criteria
The treatment is indicated for pediatric patients with severe Leukocyte Adhesion Deficiency I (LAD-I) caused by biallelic variants in the ITGB2 gene, specifically for those without an available human leukocyte antigen (HLA)-matched sibling donor for allogeneic hematopoietic stem cell transplant.
Selection Process
The approval and subsequent requirements are based on the FDA’s evaluation. Continued approval may be contingent upon the verification of clinical benefit in confirmatory trials conducted by the company.
Official Notification & Links
Important Instructions
- Severe LAD-I is a rare, inherited immune deficiency caused by mutations in the ITGB2 gene.
- Patients with severe LAD-I experience recurrent, life-threatening bacterial and fungal infections.
- Kresladi consists of the patient’s own hematopoietic (blood) stem cells, which are genetically modified.
- The treatment involves a single dose infused intravenously following conditioning.
- Rocket Pharmaceuticals, Inc. must conduct post-marketing requirements to confirm clinical benefit.
- Common side effects include anemia, low platelet and white blood cell counts, mouth sores, and various infections.
Conclusion
This accelerated approval provides a breakthrough treatment option for pediatric patients with severe LAD-I, addressing the underlying cause of this serious and rare disease.
