US FDA declines to approve Rocket Pharma's immune disorder gene therapy

[June 28, 2024]  (Reuters) -The U.S. Food and Drug Administration has declined to approve Rocket Pharmaceuticals' gene therapy to treat a rare and severe pediatric disorder that causes the immune system to malfunction, the company said on Friday.

The FDA, through a so-called "complete response letter", requested limited additional information related to certain processes for the gene therapy to complete its review.

Rocket was seeking approval from the agency to use the therapy, called Kresladi, in treating patients with severe Leukocyte Adhesion Deficiency-I (LAD-I).

In these patients, the white blood cells cannot stick properly to blood vessel walls of an infection site due to a mutation in the protein-encoding ITGB2 gene. This could lead to severe immune system deficiencies and recurrent infections.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo

(Reporting by Pratik Jain in Bengaluru; Editing by Krishna Chandra Eluri and Devika Syamnath)

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