FDA Approves CAR T-cell Therapy for Adults with Lymphoma

Gilead Science’s 28 August purchase of Kite Pharma, Inc., one of the companies developing CAR T-cells, a therapy that harnesses the body’s own immune system to recognize and attack malignant cells, paid off in spades last week. On Wednesday, 18 October, the US Food and Drug Administration (FDA) approved Kite’s Yescarta (axicabtagene ciloleucel) for the treatment of adult patients witBreakthrough for Pharma Industr17h relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma, and DLBCL arising from follicular lymphoma (transformed follicular lymphoma).

The FDA approved Kymriah (tisagenlecleucel, CTL019), the Novartis CAR T-cell therapy for the treatment of pediatric and young-adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia, on 30 August 2017.

Perhaps the most extraordinary medical treatment humankind has seen thus far, CAR T-cell therapy promises to transform both the pharmaceutical industry and patients’ lives. The price tags remain steep: $373,000 (Kite) and $475,000 (Novartis), as both therapies carry high manufacturing costs. Standard therapies, such as bone marrow transplants, are in a similar price range.

Kite Pharma filed a Marketing Authorization Application with the European Medicines Agency for axicabtagene ciloleucel in July 2017, the first CAR T-cell application in Europe. Potential approval is expected in the first half of 2018. Yescarta has received Priority Medicines regulatory support for DLBCL in the European Union.  

To learn more about this exciting new therapy, the patients it benefits, and the industry it seeks to transform, join us in San Francisco from 4 to 6 December at the ISPE 2017 Biopharmaceutical Manufacturing Conference, where Gilead and Kite will deliver the opening keynote address.

In addition to the cover story, check out the Special Report: Setting the Course for Biopharma's Future featuring the following articles:

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  Features

  Breakthrough for Pharma Industry & Patients - FDA Approved Gene Transfer Therapy

  1 September 2017

  On 30 August, the US Food and Drug Administration (FDA) approved Novartis's Kymriah (tisagenlecleucel, CTL019), the first gene therapy for the treatment of pediatric and young-adult patients with relapsed and refractory B-cell acute lymphoblastic leukemia (ALL). Kymriah belongs to a class of drugs called chimeric antigen receptor (CAR) T-cell therapies, which constitute a form of immunotherapy...

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  Special Reports

  Proof of Closure: Life Cycle of Closed Systems

  1 September 2017

  This article was developed by members of the ISPE Biotechnology Community of Practice. The views and opinions are those of the authors and do not necessarily reflect the official policy or position of Hargrove Life Sciences, M+W, Novartis, Sandoz, Takeda, or any of their officers.

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  Special Reports

  China & India Target Future GMP Manufacturing

  1 September 2017
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  Special Reports

  Engineer's Role in the Biopharmaceutical Supply Chain

  1 September 2017

  As the industry evolves in its quest to increase quality, productivity, efficiency, and safety, engineers must be equipped with skills that enable them to meet new and challenging demands. This first in a two-part series focused on biopharmaceutical manufacturing explores a few of the exciting roles engineers play to drive continuous improvement. Part 2, slated for Pharmaceutical Engineering's...