BLA 125646
tisagenlecleucel
Novartis Pharmaceuticals Corporation
Reviewers
Xiaobin Victor Lu, Ph.D Andrew Byrnes, Ph.D. Kimberly Schultz, Ph.D. Elena Gubina, Ph.D Thomas Finn, Ph.D.
BC Reviewer Denise Gavin, Ph.D. Gene Therapies Branch
Division of Cellular and Gene Therapies
BLA #: 125646/0 USAN name: tisagenlecleucel CMC Review
1.
BLA#: STN 125646
REVIEW DATE: August 29, 2017
PRIMARY REVIEW TEAM:
Medical Officer: Maura O’Leary, MD, and Donna Przepiorka, MD, PhD Pharm/Tox: Ying Huang, PhD
Product Quality Team: Xiaobin (Victor) Lu, PhD, Andrew Byrnes, PhD, Kimberly Schultz, 2.
3.
PhD, Elena Gubina, PhD, and Tom Finn, PhD
Facilities: Randa Melhem, PhD, Joan Johnson, MS, Statistics: Xue (Mary) Lin, PhD Labeling: Dana Jones, PharmD RPM: Erica Giordano
4.
COMMUNICATIONS WITH APPLICANT:
Communication/Document Date
Teleconference/batch analysis data in electronic format 2/24/2017 Teleconference/CMC discussion with the applicant to clarify 3/2/2017 established conditions
Teleconference /discuss the CMC section of the AC meeting 5/3/2017 Teleconference/Mycoplasma Testing at (b) (4) 5/31/2017 Teleconference/ FMO control/ lot release specification/ Plans for 7/6/2017 OOS lots/VCN/appearance test/
Teleconference/(b) (4)samples at (b) (4)
deviation GMP2491: storage of sterility 07/17/2017
.
5.
SUBMISSION(S) REVIEWED:
Submission Date Received Review Completed (Yes/No)
Original submission
2/2/2017 Yes Amendment #6 3/14/2017 Yes Amendment #8 3/15/2017 Yes Amendment #12 4/3/2017 Yes Amendment #14 4/7/2017 Yes Amendment #15 4/14/2017 Yes Amendment #18 4/27/2017 Yes Amendment #20 5/1/2017 Yes Amendment #23 5/5/2017 Yes Amendment #24 5/10/2017 Yes Amendment #25 5/11/2017 Yes Amendment #26 5/12/2017 Yes Amendment #30 5/30/2017 Yes Amendment #31
6/6/2017
Yes
2
BLA #: 125646/0 Amendment #32 Amendment #34 Amendment #36 Amendment #39 Amendment #42 Amendment #44 Amendment #45 Amendment #46 Amendment #48 Amendment #50 Amendment #51 Amendment #52 Amendment#55 Amendment#56 Amendment#57 Amendment#61
USAN name: tisagenlecleucel 6/16/2017 6/21/2017 6/21/2017 7/7/2017 7/11/2017 7/18/2017 7/25/2017 7/25/2017 8/2/2017 8/4/2017 8/10/2017 8/11/2017 8/23/2017 8/25/2017 8/28/2017 8/28/2017
Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes Yes
6.
DRUG PRODUCT NAME/CODE/TYPE:
a. Proprietary Name: KYMRIAH b. Trade Name: KYMRIAH
c. Non-Proprietary/USAN: tisagenlecleucel d. CAS name: Not established
e. Common name: Chimeric antigen receptor (CAR) T cells against CD19 (CAR-19) f. INN Name: tisagenlecleucel
g. Compendial Name: Not established h. OBP systematic name: N/A i. Other Names: CTL019
j. FDA UNII number: Q6C9WHR03O PHARMACOLOGICAL CATEGORY: CD19-directed genetically modified autologous T cell immunotherapy
DOSAGE FORM: Cell suspension
STRENGTH/POTENCY: Up to 2.5 x 108 CAR positive viable T cells ROUTE OF ADMINISTRATION: Intravenous infusion
INSPECTIONAL ACTIVITIES: PLI inspections completed 5/25/2017 CONSULTS REQUESTED: N/A PRECEDENTS: First-in-class ADMINISTRATIVE
7. 8. 9. 10. 11.
14. 16. 17.
3
诺华CAR-T疗法KYMRIAH申报CMC部分
