Novartis CAR-T Therapy Receives FDA Breakthrough Therapy Designation

Medical Supporter first learned about CAR-T around late 2014, when a friend with colorectal cancer asked whether this therapy was available in Japan. At the time, there was little related information available, and Japan did not yet have the relevant technology. Subsequently, clinical trials began emerging — for example, Jichi Medical University began recruiting volunteers. It was through the data they released that we gained some understanding of this therapy. In 2014, the FDA also designated it as a Breakthrough Therapy for B-cell acute lymphoblastic leukemia (ALL) — a blood disease caused by abnormal proliferation of lymphocytes in the body.

Here we share the news from April 18, 2017: The U.S. FDA announced Breakthrough Therapy Designation (BTD) for CTL019 (tisagenlecleucel-T), a CAR-T therapy, for adult patients with relapsed/refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who had received two or more prior systemic therapies. In March of that year, CTL019 also received Breakthrough Therapy Designation for use in pediatric and young adult patients with DLBCL.

The FDA Breakthrough Therapy Designation was based on the JULIET international Phase 2 clinical trial (NCT02445248) in adult patients with relapsed or refractory DLBCL. The results of this trial will be published at future medical meetings.

*1: The Breakthrough Therapy Designation system originated in 2013, when the U.S. FDA introduced a pathway to certify therapies and drugs with exceptional efficacy and breakthrough potential, accelerating their development. A key benefit is that receiving such designation attracts significantly more investment during the R&D process, providing more resources to accelerate outcomes.

*2: CAR-T cell therapy is a new type of immunotherapy that is individually customized for each patient. In simple terms, the T cells in the patient's blood are genetically engineered to express a receptor targeting a specific antigen, enabling them to attack B cells.

A diagram illustrating the process is available in Novartis's press release from December 4, 2015: http://www.novartispharmaceuticals.com/en/stories/detail/a-new-kind-of-clinical-trial

*3: The original press release: https://www.novartis.com/news/media-releases/novartis-car-t-cell-therapy-ctl019-receives-fda-breakthrough-therapy-designation

Medical Supporter was formerly certified as an international medical visa guarantor by Japan's Ministry of Foreign Affairs and the Ministry of Economy, Trade and Industry (B-066).

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Related Cancer Information

• Colorectal cancer