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    1. Development
    2. Drug Approvals

    FDA rejects Rocket gene therapy

    July 1, 2024

    After extending the review period for three months, the U.S. FDA has now issued a complete response letter in response to Rocket Pharmaceuticals' BLA for Kresladi, a lentiviral vector-based gene therapy to treat severe leukocyte adhesion deficiency-I (LAD-I). 
     
    Accoding to Rocket, per the CRL, the agency requested limited additional chemistry manufacturing and controls (CMC) information to complete its review. The drugmaker met with FDA senior leaders from CBER to align on the scope of additional CMC information needed to support the approval of Kresladi as quickly as possible.

    Kresladi (marnetegragene autotemcel) originally had a PDUFA date of March 31 for LAD-I, a rare pediatric immunodeficiency disorder, but FDA made the call in February to push the review date to June 30 to allow additional time to review clarifying CMC  information submitted by Rocket in response to the agency's information requests.

    LAD-1 is caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18, a key protein needed to combat infections. Rocket's Kresladi contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18.

    The NJ-based biotech is hopeful that the therapy has the potential to change the treatment paradigm for patients living with severe LAD-I — one of the most aggressive and highly fatal immunodeficiencies ever characterized. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon, according to Rocket.