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

Rocket Pharmaceuticals

28/06/2024 12:36

(Adds details throughout)
       June 28 (Reuters) - The U.S. Food and Drug
Administration has declined to approve Rocket Pharmaceuticals'
 RCKT.O  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.  
  

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