FDA Approves Kymriah for Cancer and Life-threatening Diseases Treatment

Author: U.S. Food and Drug Administration
Published: 2017/09/22 - Updated: 2025/05/14
Publication Details: Peer-Reviewed, Announcement
Topic: FDA News and Approvals - Publications List

Page Content: Synopsis - Introduction - Main - Insights, Updates

Synopsis: This report details the U.S. Food and Drug Administration's historic approval of Kymriah (tisagenlecleucel), the first gene therapy authorized in the United States for treating certain pediatric and young adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). Kymriah is a personalized, cell-based immunotherapy that uses a patient's own T-cells, genetically engineered to target and destroy cancer cells bearing the CD19 antigen.

The approval is authoritative, reflecting rigorous clinical evaluation: in a multicenter trial, 83% of patients achieved remission within three months. This breakthrough offers a crucial new option for individuals-especially children and young adults-whose disease has not responded to standard treatments, addressing a significant unmet medical need. The report also highlights the therapy's risks, including potentially severe side effects like cytokine release syndrome and neurological events, and describes the FDA's requirements for specialized hospital certification and risk management to ensure patient safety.

Such innovation is especially relevant for people with disabilities, seniors, or those facing life-threatening diseases, as it expands the landscape of available treatments where few alternatives exist and underscores the FDA's commitment to advancing safe, effective therapies - Disabled World (DW).

Introduction

The U.S. Food and Drug Administration has issued a historic action making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

Focus

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines, and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL).

"We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," said FDA Commissioner Scott Gottlieb, M.D. "New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we're committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving."

Kymriah, a cell-based gene therapy, is approved in the United States for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is a genetically-modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient's own T-cells, a type of white blood cell known as a lymphocyte. The patient's T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.

ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S. The National Cancer Institute estimates that approximately 3,100 patients aged 20 and younger are diagnosed with ALL each year. ALL can be of either T- or B-cell origin, with B-cell the most common. Kymriah is approved for use in pediatric and young adult patients with B-cell ALL and is intended for patients whose cancer has not responded to or has returned after initial treatment, which occurs in an estimated 15-20 percent of patients.

"Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER). "Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials."

The safety and efficacy of Kymriah were demonstrated in one multicenter clinical trial of 63 pediatric and young adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was 83 percent.

Treatment with Kymriah has the potential to cause severe side effects. It carries a boxed warning for cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T-cells causing high fever and flu-like symptoms, and for neurological events. Both CRS and neurological events can be life-threatening. Other severe side effects of Kymriah include serious infections, low blood pressure (hypotension), acute kidney injury, fever, and decreased oxygen (hypoxia). Most symptoms appear within one to 22 days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also destroy those normal B cells that produce antibodies, there may be an increased risk of infections for a prolonged period of time.

The FDA also expanded the approval of Actemra (tocilizumab) to treat CAR T-cell-induced severe or life-threatening CRS in patients 2 years of age or older. In clinical trials in patients treated with CAR-T cells, 69 percent of patients had complete resolution of CRS within two weeks following one or two doses of Actemra.

Because of the risk of CRS and neurological events, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use (ETASU). The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified. As part of that certification, staff involved in the prescribing, dispensing, or administering of Kymriah are required to be trained to recognize and manage CRS and neurological events. Additionally, the certified health care settings are required to have protocols in place to ensure that Kymriah is only given to patients after verifying that tocilizumab is available for immediate administration. The REMS program specifies that patients be informed of the signs and symptoms of CRS and neurological toxicities following infusion - and of the importance of promptly returning to the treatment site if they develop fever or other adverse reactions after receiving treatment with Kymriah.

To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah.

The FDA granted Kymriah Fast Track, Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency approach. The clinical review was coordinated by the FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

The FDA granted approval of Kymriah to Novartis Pharmaceuticals Corp. The FDA granted the expanded approval of Actemra to Genentech Inc.

Insights, Analysis, and Developments

Editorial Note: The FDA's approval of Kymriah marks a pivotal moment in cancer care, not only for its scientific innovation but for the hope it offers to families confronting the most daunting diagnoses. While the therapy's complexity and risks demand careful oversight, its demonstrated ability to induce remission in patients with few remaining options is a testament to the progress of personalized medicine. As gene and cell therapies continue to evolve, their impact on rare and aggressive diseases could redefine expectations for survival and quality of life, particularly for vulnerable populations who have historically had limited access to transformative treatments - Disabled World (DW).

Attribution/Source(s): This peer reviewed publication was selected for publishing by the editors of Disabled World (DW) due to its relevance to the disability community. Originally authored by U.S. Food and Drug Administration and published on 2017/09/22, this content may have been edited for style, clarity, or brevity. For further details or clarifications, U.S. Food and Drug Administration can be contacted at fda.gov NOTE: Disabled World does not provide any warranties or endorsements related to this article.

Explore Similar Topics

CAR-T Cell Therapy: Cancer Treatment Through Precision Immunotherapy: An in-depth look at CAR-T cell therapy, a personalized cancer treatment offering targeted, long-term immune response for hard-to-treat blood cancers.

T Cell Therapy: Fighting Cancer and Infections: T cell immunity has evolved to recognize and respond to health threats and provide a lifelong memory that prevents recurrent disease.

Personalized Medicine, Pharmacogenomics and Human Genome: Personalized medicine is moving us closer to more precise, predictable and powerful medicine, customized for the individual patient.

Immunotherapy Treatment Options and Information: Immunotherapy or a regimen of allergy shots is treatment designed to alter the immune system responses to substances they are allergic to.

: Cognoa ASD Diagnosis Aid is an aid for diagnosis of ASD in patients 18 months - 5 years of age who are at risk of developmental delay based on concerns of a parent, caregiver, or health care provider.

: U.S. FDA authorizes marketing of Neurolutions IpsiHand Upper Extremity Rehabilitation System to facilitate muscle rehabilitation in stroke patients.

Citing and References

Founded in 2004, Disabled World (DW) is a leading resource on disabilities, assistive technologies, and accessibility, supporting the disability community. Learn more on our About Us page.

Cite This Page: U.S. Food and Drug Administration. (2017, September 22 - Last revised: 2025, May 14). FDA Approves Kymriah for Cancer and Life-threatening Diseases Treatment. Disabled World (DW). Retrieved May 23, 2025 from www.disabled-world.com/medical/pharmaceutical/fda/kymriah.php

Permalink: <a href="https://www.disabled-world.com/medical/pharmaceutical/fda/kymriah.php">FDA Approves Kymriah for Cancer and Life-threatening Diseases Treatment</a>: FDA approves Kymriah, the first U.S. gene therapy, offering new hope for children and young adults with hard-to-treat acute lymphoblastic leukemia.

While we strive to provide accurate and up-to-date information, it's important to note that our content is for general informational purposes only. We always recommend consulting qualified healthcare professionals for personalized medical advice. Any 3rd party offering or advertising does not constitute an endorsement.