Rocket Pharmaceuticals’ gene therapy Kresladi has been hit with an FDA Complete Response Letter requesting additional chemistry, manufacturing and controls information to complete its review.
Rocket Pharmaceuticals announced Friday that the FDA rejected its rare disease gene therapy Kresladi (marnetegragene autotemcel) in a Complete Response Letter requesting “limited additional” chemistry, manufacturing and controls information to complete the regulator’s review.
Kresladi, a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I), has already faced delays from the agency as it was initially supposed to find out its fate in March 2024. However, the FDA pushed back the PDUFA date to June 30.
Rocket said it has met with leaders from the FDA’s Center for Biologics Evaluation and Research (CBER) to “align on the limited scope” of additional chemistry, manufacturing and controls (CMC) information and support Kresladi’s approval as soon as possible. However, Rocket’s stock price fell by over 11% in premarket trading Friday.
“It is reassuring to have the FDA as a close collaborator who understands the high unmet medical need, clear clinical benefit and importance of timely patient access,” Rocket CEO Gaurav Shah said in a statement. “CBER leadership’s direct involvement and commitment to working expeditiously to deliver this therapy to patients gives us great hope on behalf of the primary immunodeficiency community.”
Rocket’s gene therapy is designed to treat LAD-I, a rare pediatric disease caused by mutations in the ITGB2 gene. This mutation leads to a faulty CD18 protein, allowing blood vessels to combat infection, and can cause severe bacterial and fungal infections in patients starting from birth.
Kresladi’s Biologics License Application (BLA) was accepted in October last year based on Phase I/II data showing a 100% overall survival rate at 12 months post-infusion and the entire follow-up duration for nine LAD-I patients. The data also showed decreases in the incidence of infections, reductions of skin lesions and the return of wound repair capabilities.
Rocket joins other companies this week who have received CRLs from the FDA citing manufacturing issues. Merck and Daiichi Sankyo announced Thursday that its antibody-drug conjugate for non-small cell lung cancer, patritumab deruxtecan (HER3-DXd), was given a CRL related to findings at a third-party manufacturing facility. The FDA also on Tuesday denied AbbVie’s New Drug Application for its Parkinson’s disease candidate ABBV-951, citing observations from an inspection at a third-party manufacturer.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
