FDA denies Rocket gene therapy

Kresladi (marnetegragene autotemcel), a treatment initially set for a PDUFA review date of March 31 for Leukocyte Adhesion Deficiency-I (LAD-I), saw its assessment deadline extended to June 30 by the FDA in February. This extension was to allow more time for reviewing additional clarifying CMC (chemistry, manufacturing, and controls) information provided by Rocket Pharmaceuticals in response to the agency's queries.

LAD-I, a rare pediatric immunodeficiency disorder, stems from mutations in the ITGB2 gene, responsible for encoding the beta-2 integrin component CD18. CD18 is a crucial protein in the body's defense against infections. Rocket’s Kresladi involves genetically modifying autologous hematopoietic stem cells using a lentiviral vector to introduce a functional copy of the ITGB2 gene, thereby restoring the production of the beta-2 integrin component CD18.

Rocket Pharmaceuticals, headquartered in New Jersey, is optimistic about Kresladi's potential to revolutionize the treatment landscape for patients suffering from severe LAD-I—a particularly severe and often fatal immunodeficiency. According to the company, without a successful bone marrow transplant, the mortality rate for children with severe LAD-I ranges between 60-75% before they reach the age of 2, and survival beyond the age of 5 is rare.