7/12/22, 3:48 PM
`
`FDA approval brings first gene therapy to the United States | FDA
`
`FDA NEWS RELEASE
`FDA approval brings rst gene therapy to the United States
`
`For Immediate Release:
`August 30, 2017
`
`This release was updated on Aug. 30, 2017 to correctly identify the FDA designations granted to Kymriah.
`
`Español (/news-events/comunicados-de-prensa/primera-terapia-genetica-en-los-estados-unidos-es-aprobada-por-la-fda)
`
`The U.S. Food and Drug Administration issued a historic action today 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.
`
`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.
`
`https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states
`
`1/4
`
`UPenn Ex. 2007
`Miltenyi v. UPenn
`IPR2022-00855
`
`

`

`FDA approval brings first gene therapy to the United States | FDA
`7/12/22, 3:48 PM
`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 today 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
`
`https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states
`
`2/4
`
`UPenn Ex. 2007
`Miltenyi v. UPenn
`IPR2022-00855
`
`

`

`FDA approval brings first gene therapy to the United States | FDA
`7/12/22, 3:48 PM
`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 Priority Review (/patients/fast-track-breakthrough-therapy-
`accelerated-approval-and-priority-review/priority-review) and Breakthrough Therapy
`(/patients/fast-track-breakthrough-therapy-accelerated-approval-and-priority-
`review/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.
`
`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.
`
`Related Information
`
`FDA: Cellular and Gene Therapy Products (/cellular-gene-therapy-products)
`
`FDA: Oncology Center of Excellence (/oncology-center-excellence)
`
`NIH: Childhood Acute Lymphoblastic Leukemia
`(https://www.cancer.gov/types/leukemia/patient/child-all-treatment-pdq)
`
`FDA: What is Gene Therapy? (/vaccines-blood-biologics/cellular-gene-therapy-
`products/what-gene-therapy)
`
`FDA: Kymriah (tisagenlecleucel) product page (/vaccines-blood-biologics/approved-
`products/kymriah-tisagenlecleucel)
`
`FDA: Approved Cellular and Gene Therapy Products (/approved-products-1)
`
`
`
`https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states
`
`3/4
`
`UPenn Ex. 2007
`Miltenyi v. UPenn
`IPR2022-00855
`
`

`

`FDA approval brings first gene therapy to the United States | FDA
`7/12/22, 3:48 PM
`CAR T-cell therapy approved to treat certain children and young adults with B-cell acute
`lymphoblastic leukemia
`
`Media
`
` Andrea Fischer (mailto:andrea.fischer@fda.hhs.gov)
` 301-796-0393
`
` Angela Stark (mailto:angela.stark@fda.hhs.gov)
` 301-796-0397
`
`Consumers
`
` 888-INFO-FDA, OCOD@fda.hhs.gov (mailto:OCOD@fda.hhs.gov)
`
`###
`
` More Press Announcements (/news-events/newsroom/press-announcements)
`
`https://www.fda.gov/news-events/press-announcements/fda-approval-brings-first-gene-therapy-united-states
`
`4/4
`
`UPenn Ex. 2007
`Miltenyi v. UPenn
`IPR2022-00855
`
`