FDA Requests More Data From Rocket for Kresladi Gene Therapy

The FDA has issued a complete response letter to Rocket Pharmaceuticals requesting additional chemistry, manufacturing and controls information before reviewing the company’s BLA for Kresladi (marnetegragene autotemcel).

Kresladi is a lentiviral vector-based gene therapy that contains autologous (patient-derived) hematopoietic stem cells intended to treat severe leukocyte adhesion deficiency-I (LAD-I).

The company reported that top-line data from a global phase 1/2 study of Kresladi demonstrated 100 percent overall survival at 12 months post-infusion (and for the entire duration of follow up) for all nine LAD-I patients with 18 to 42 months of available follow up.

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