FDA Grants Priority Review to Rocket Pharma's Groundbreaking Gene Therapy for Rare LAD-I

In a momentous development for patients suffering from Leukocyte Adhesion Deficiency-I (LAD-I), Rocket Pharmaceuticals (NASDAQ: RCKT) has announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for RP-L201, a potentially life-saving gene therapy.

Further underscoring its significance, the FDA has granted RP-L201 Priority Review status, setting a Prescription Drug User Fee Act (PDUFA) target action date of March 31, 2025.

This pivotal decision brings the scientific community and affected families a step closer to what could be the first approved therapy for severe LAD-I, a devastating and ultra-rare genetic disorder.

LAD-I is caused by mutations in the ITGB2 gene, which leads to a critical deficiency in the CD18 integrin protein. This protein is essential for white blood cells (leukocytes) to exit the bloodstream and fight infections, leaving patients highly vulnerable to severe, recurrent bacterial and fungal infections.

Without effective treatment, the disease often results in early childhood mortality, with bone marrow transplantation (BMT) being the only current curative option, though it carries significant risks and is not always feasible.

Rocket Pharma's RP-L201 is designed to directly address the root cause of LAD-I.

It is an investigational lentiviral vector-based gene therapy that aims to deliver a functional copy of the ITGB2 gene to a patient's own hematopoietic stem cells. By correcting the genetic defect, the therapy intends to restore the production of functional CD18, thereby enabling the immune system to properly combat infections and improving patient outcomes.

The BLA submission was supported by compelling and robust clinical data from ongoing global studies, demonstrating the therapy's potential to provide sustained clinical benefit and a dramatically improved quality of life for those afflicted.

The FDA's decision to grant Priority Review is a testament to the urgent unmet medical need for LAD-I patients and the promising data generated by RP-L201.

Priority Review is reserved for applications for therapies that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions. This designation shortens the FDA's review timeline from the standard 10 months to 6 months, accelerating the potential availability of this innovative treatment.

Adding to its expedited pathway, RP-L201 has already received several crucial designations from the FDA, including Orphan Drug, Rare Pediatric Disease, and Regenerative Medicine Advanced Therapy (RMAT) designations.

These designations highlight the therapy's potential to address a rare disease with limited treatment options and facilitate a more rapid development and review process.

The acceptance of the BLA and the granting of Priority Review for RP-L201 represents a monumental stride forward in gene therapy and for the LAD-I community.

It signifies a beacon of hope for patients and their families, promising a future where a single-administration treatment could offer a lasting solution to a debilitating disease, potentially transforming the lives of those who previously faced a grim prognosis. All eyes will now be on the FDA as the PDUFA date approaches, eagerly anticipating a decision that could usher in a new era of treatment for LAD-I.