FDA approval brings very first gene therapy to the United States
CAR T-cell therapy approved to treat certain children and youthful adults with B-cell acute lymphoblastic leukemia
For Instant Release
Release
The U.S. Food and Drug Administration issued a historic activity today making the very first gene therapy available in the United States, ushering in a fresh treatment to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthful adult patients with a form of acute lymphoblastic leukemia (ALL).
«We`re injecting a fresh frontier in medical innovation with the capability to reprogram a patient`s own cells to attack a deadly cancer,» said FDA Commissioner Scott Gottlieb, M.D. «Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability 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 twenty five years of age with B-cell precursor ALL that is refractory or in 2nd 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 fresh 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 figure 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 Three,100 patients aged twenty and junior 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 youthful 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 treatment that fills an significant unmet need for children and youthfull 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 fresh 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 sixty three pediatric and youthful adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was eighty three 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 show up within one to twenty two days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also demolish those normal B cells that produce antibodies, there may be an enhanced 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 two years of age or older. In clinical trials in patients treated with CAR-T cells, sixty nine percent of patients had finish 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 instant 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 investigate involving patients treated with Kymriah.
The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency treatment. 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.
FDA approval brings very first gene therapy to the United States
FDA approval brings very first gene therapy to the United States
CAR T-cell therapy approved to treat certain children and youthfull adults with B-cell acute lymphoblastic leukemia
For Instant Release
Release
The U.S. Food and Drug Administration issued a historic activity today making the very first gene therapy available in the United States, ushering in a fresh treatment to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthfull adult patients with a form of acute lymphoblastic leukemia (ALL).
«We`re injecting a fresh frontier in medical innovation with the capability to reprogram a patient`s own cells to attack a deadly cancer,» said FDA Commissioner Scott Gottlieb, M.D. «Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability 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 twenty five years of age with B-cell precursor ALL that is refractory or in 2nd 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 fresh 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 bod 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 Three,100 patients aged twenty and junior 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 youthfull 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 treatment that fills an significant unmet need for children and youthful 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 fresh 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 sixty three pediatric and youthfull adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was eighty three 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 emerge within one to twenty two days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also demolish those normal B cells that produce antibodies, there may be an enlargened 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 two years of age or older. In clinical trials in patients treated with CAR-T cells, sixty nine percent of patients had accomplish 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 instant 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 explore involving patients treated with Kymriah.
The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency treatment. 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.
FDA approval brings very first gene therapy to the United States
FDA approval brings very first gene therapy to the United States
CAR T-cell therapy approved to treat certain children and youthfull adults with B-cell acute lymphoblastic leukemia
For Instantaneous Release
Release
The U.S. Food and Drug Administration issued a historic activity today making the very first gene therapy available in the United States, ushering in a fresh treatment to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthful adult patients with a form of acute lymphoblastic leukemia (ALL).
«We`re coming in a fresh frontier in medical innovation with the capability to reprogram a patient`s own cells to attack a deadly cancer,» said FDA Commissioner Scott Gottlieb, M.D. «Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability 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 twenty five years of age with B-cell precursor ALL that is refractory or in 2nd 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 fresh 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 bod 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 Trio,100 patients aged twenty and junior 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 youthful 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 treatment that fills an significant unmet need for children and youthfull 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 fresh 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 sixty three pediatric and youthfull adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was eighty three 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 show up within one to twenty two days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also demolish those normal B cells that produce antibodies, there may be an enhanced 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 two years of age or older. In clinical trials in patients treated with CAR-T cells, sixty nine percent of patients had accomplish 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 instant 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 examine involving patients treated with Kymriah.
The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency treatment. 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.
FDA approval brings very first gene therapy to the United States
FDA approval brings very first gene therapy to the United States
CAR T-cell therapy approved to treat certain children and youthful adults with B-cell acute lymphoblastic leukemia
For Instantaneous Release
Release
The U.S. Food and Drug Administration issued a historic activity today making the very first gene therapy available in the United States, ushering in a fresh treatment to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthful adult patients with a form of acute lymphoblastic leukemia (ALL).
«We`re injecting a fresh frontier in medical innovation with the capability to reprogram a patient`s own cells to attack a deadly cancer,» said FDA Commissioner Scott Gottlieb, M.D. «Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability 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 twenty five years of age with B-cell precursor ALL that is refractory or in 2nd 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 fresh 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 bod 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 Three,100 patients aged twenty and junior 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 youthfull 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 treatment that fills an significant unmet need for children and youthfull 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 fresh 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 sixty three pediatric and youthful adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was eighty three 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 emerge within one to twenty two days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also demolish those normal B cells that produce antibodies, there may be an enlargened 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 two years of age or older. In clinical trials in patients treated with CAR-T cells, sixty nine percent of patients had accomplish 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 instant 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 explore involving patients treated with Kymriah.
The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency treatment. 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.
FDA approval brings very first gene therapy to the United States
FDA approval brings very first gene therapy to the United States
CAR T-cell therapy approved to treat certain children and youthfull adults with B-cell acute lymphoblastic leukemia
For Instantaneous Release
Release
The U.S. Food and Drug Administration issued a historic act today making the very first gene therapy available in the United States, ushering in a fresh treatment to the treatment of cancer and other serious and life-threatening diseases.
The FDA approved Kymriah (tisagenlecleucel) for certain pediatric and youthful adult patients with a form of acute lymphoblastic leukemia (ALL).
«We`re injecting a fresh frontier in medical innovation with the capability to reprogram a patient`s own cells to attack a deadly cancer,» said FDA Commissioner Scott Gottlieb, M.D. «Fresh technologies such as gene and cell therapies hold out the potential to convert medicine and create an inflection point in our capability 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 twenty five years of age with B-cell precursor ALL that is refractory or in 2nd 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 fresh 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 assets 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 Trio,100 patients aged twenty and junior 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 youthfull 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 treatment that fills an significant unmet need for children and youthfull 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 fresh 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 sixty three pediatric and youthfull adult patients with relapsed or refractory B-cell precursor ALL. The overall remission rate within three months of treatment was eighty three 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 show up within one to twenty two days following infusion of Kymriah. Since the CD19 antigen is also present on normal B-cells, and Kymriah will also ruin those normal B cells that produce antibodies, there may be an enlargened 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 two years of age or older. In clinical trials in patients treated with CAR-T cells, sixty nine percent of patients had accomplish 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 instantaneous 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 examine involving patients treated with Kymriah.
The FDA granted Kymriah Priority Review and Breakthrough Therapy designations. The Kymriah application was reviewed using a coordinated, cross-agency treatment. 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.