Prateek Jain

LONDON: Rocket Pharmaceuticals said on Friday that the US Health regulators refused to approve it gene therapy For treatment Rare and serious Pediatric disorders It causes malfunction in the immune system, causing its shares to fall more than 12% in early trade.

The Food and Drug Administration has sought limited additional information related to certain procedures for gene therapy through a so-called ‘complete response letter.’ Cresladto complete its review.

The company was seeking its first approval from the agency for the use of this therapy to treat patients suffering from severe leukocyte adhesion deficiency-I (Leukocyte Adhesion Deficiency-I).LAD-I), Rockett estimates the disease affects about 800 to 1,000 people in the US and Europe.

In these patients, mutations in the protein-encoding ITGB2 gene cause white blood cells to fail to stick properly to the blood vessel walls of the infection site. This can lead to severe immune deficiency and frequent infections.

The New Jersey-based company said it FDA To discuss the information sought for quick approval.

The company’s marketing approval application was based on data from an early to mid-stage trial in which all nine patients survived at least 12 months following injection.

In February, the FDA extended its deadline for deciding on approval for gene therapy to review similar data related to certain procedures.

Analysts at TD Cowen said then that the delay was a problem related to FDA bandwidth, not an issue on the company’s part.

TD Cowen analyst Tyler Van Buren said Cresaldi is a small opportunity for Rocket, but success here would provide validation to the company’s lentiviral platform, which is being used for other treatments in the pipeline with large patient populations.

The only treatment currently for the condition is a bone marrow transplant, which is often not readily available and carries considerable risks. (Reporting by Pratik Jain in Bengaluru; Editing by Devika Shyamnath, Shailesh Kuber and Sriraj Kalluvilla)