FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Kymriah (tisagenlecleucel)

Indication:

Treatment of patients aged <25 years with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse.

Mechanism:

CD19-directed genetically modified autologous T cell immunotherapy. A patient’s own T cells are reprogrammed with a transgene encoding a chimeric antigen receptor (CAR) to identify and eliminate CD19-expressing malignant and normal cells.

Dosage:

A single dose of tisagenlecleucel is administered by IV infusion 2-14 days after completing lymphodepletion with conditioning chemotherapy.

≤50 kg: 0.2-5 x 10⁶ CAR-positive viable T cells/kg

>50 kg: 0.1-2.5 x 10⁸ CAR-positive viable T cells/kg

See the Certificate of Analysis for the actual number of CAR-positive T cells in the product.

Approval:

Approval was based on results of the ELIANA trial, an open-labeled, multicenter, single arm phase 2 study. Among the 63 evaluable patients, 52 achieved complete remission (CR) or CR with incomplete blood count recovery (CRi) within 3 months of infusion, all of whom were associated with minimal residual disease-negative status in the bone marrow. The relapse-free probability at 6 months after remission was 75%, and the probability of survival was 89% at 6 months and 79% at 12 months, respectively.

Reference:

Buechner J, et al. Global registration trial of efficacy and safety of CTL019 in pediatric and young adult patients with relapsed/refractory (R/R) acute lymphoblastic leukemia (ALL): Update to the interim analysis [abstract]. Presented at the European Hematology Association Annual Meeting. June 24, 2017;Abstract S476.

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Cite this: Mary L Windle. FDA New Drug and Biologic Approvals -- 2017 Year-in-Review - Medscape - Jan 11, 2018.