FDA Panel Votes Unanimously in Favor of First CAR-T Cancer Therapy

Regulatory NewsRegulatory News | 12 July 2017 |  By 

The US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee on Wednesday voted unanimously, 10 to 0, in favor of the benefit-risk profile for the first of a new kind of cancer therapy, known as a Chimeric Antigen Receptor T-cell (CAR-T) therapy.

Although the outside panel of experts raised questions about concerns with the safety and manufacturing of the Novartis treatment, known as CTL019 (tisagenlecleucel-T), the panel did not question the efficacy of the CAR-T therapy in relapsed and refractory (r/r) pediatric and young adult patients with B-cell acute lymphoblastic leukemia (ALL).

The National Cancer Institute’s James Gulley said in the panel discussion: "This is a product that has shown patient benefit, and I think our goal here is to try and find ways that we can make sure we have optimal safety and potency."

Other panelists called the treatment a "major advance" and that novel therapy meets a "dire, unmet need."

Novartis’ therapy has been granted a priority review designation (meaning six months for the FDA review, rather than the standard 10 months) based on a Phase II study in which 82% (41 of 50) of patients infused with CAR-T cells achieved complete remission or complete remission with incomplete blood count recovery at three months post CTL019 infusion. Novartis said Wednesday that overall survival in its pivotal trial was 89% at six months and 79% at 12 months.

Reflecting on the importance of Wednesday’s meeting, Wilson Bryan, director of FDA’s Office of Tissues and advanced therapies, said the agency recognizes there’s substantial interest among various stakeholders in the field of CAR-T and what was heard Wednesday "may be relevant and considered in evaluation of other applications in the class."

Samit Hirawat, Novartis' head of oncology global development, explained the process by which the treatment is created with patients’ own cells that are transferred from the patient, moved to a dedicated manufacturing facility in Morris Plains, New Jersey where the cells undergo enrichment and are then cryopreserved and returned to the clinical site where patients are infused.

Bryan noted at the outset of the meeting, that it’s still not clear how FDA or Novartis can assure patients that the marketed product would be the same product as what was studied in clinical trials, though Novartis will monitor patients for 15 years after treatment.


Novartis’ CTL019 is the first of many developing CAR-T therapies, which have put FDA in the unique position in which it has begun to monitor the safety events across more than 100 INDs.


Elizabeth Smith, SVP of regulatory affairs and quality at Juno Therapeutics, explained the potential for these new treatments in a comment on the Novartis meeting.

"The potential for CAR-T cells is immense. The promise of CAR-T cell therapy is evident not only in pediatric r/r ALL, but also across B Cell Malignancies, including adult ALL, aggressive r/r Non Hodgkin Lymphoma, and chronic lymphocytic leukemia. Beyond B cell malignancies, striking responses are also being seen in refractory multiple myeloma. In these heretofore intractable settings, data recently announced from Novartis, Kite Pharma, Bluebird Bio, and Juno show substantial improvements over existing therapies with unprecedented response rates and apparent long term benefi. The discussion at this ODAC may shape the development and innovation of this nascent treatment paradigm," she wrote.

Arie Belldegrun, CEO of Kite Pharma, added Wednesday: "Much of what CAR-T promises will be new. Not just to patients, but to the physicians who are evaluating these new therapies for their patients. While one-shot approaches to cancer, such as stem-cell transplantation, are an established part of the armamentarium, the idea that treatment can be given only once, in a hospital setting, is a game-changing approach."

Briefing Information for the July 12, 2017 Meeting of the Oncologic Drugs Advisory Committee 


© 2023 Regulatory Affairs Professionals Society.

Discover more of what matters to you

No taxonomy