The main efficacy outcome measures were objective response rate (ORR) and duration of response (DoR). The median time to initial response to lifileucel was 1.5 months. ORR was based on 73 subjects who received lifileucel within the recommended dosing range of 7.5 x10
9 to 72x10
9 viable cells. ORR was 31.5% (95% CI: 21.1, 43.4) and median DoR was not reached (NR) (95% CI: 4.1 months, NR).
The prescribing information contains a Boxed Warning for treatment-related mortality, prolonged severe cytopenia, severe infection, cardiopulmonary, and renal impairment. The most common adverse reactions (≥20%) in order of decreasing frequency were chills, pyrexia, fatigue, tachycardia, diarrhea, febrile neutropenia, edema, rash hypotension, alopecia, infection, hypoxia, and dyspnea. The recommended lifileucel dose is 7.5 x 10
9 to 72 x 109 viable cells.
This review used the
Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted priority review, fast track designation, Regenerative Medicine Advanced Therapy designation, and orphan drug designation. FDA expedited programs are described in the
Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s
MedWatch Reporting System or by calling 1-800-FDA-1088.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s
Project Facilitate at 240-402-0004 or email
OncProjectFacilitate@fda.hhs.gov.
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