Catherine M. Bollard, MD
Citing “unprecedented clinical success” and calling the drug “potentially paradigm-changing,” the Oncologic Drugs Advisory Committee (ODAC) voted 10-0 today to recommend approval of a biologics license application (BLA) for tisagenlecleucel (CTL019) for the treatment of patients aged 25 or younger with relapsed/refractory B-cell acute lymphoblastic leukemia (ALL).
The committee was asked to consider 4 issues:
- Manufacturing quality control with respect to identity, safety, purity and potency
- Strategies to address safety concerns including generation of replication-competent retrovirus (RCR) and insertional mutagenesis
- Risk mitigation measures for the serious risks of cytokine release syndrome and neurotoxicity
- The necessary duration patient follow-up and the type of assessments recommended
The FDA already requires 15 years of follow-up to monitor for subsequent malignant transformation for the tisagenlecleucel investigational new drug studies.
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