FDA says â€˜yesâ€™ to axicabtagene ciloleucel (Yescarta), a gene therapy for lymphoma.
Yesterday, the FDA approved Kite Pharma’s axicabtagene ciloleucel (Yescarta), a new gene therapy for adults with diffuse large B-cell lymphoma (DLBCL) who have failed to respond to or have relapsed after at least 2 other treatments, according to a press release.
Axicabtagene ciloleucel is a CAR-T cell therapy and is the second gene therapy to be approved by the FDA. This is the first gene therapy to be approved for non-Hodgkin lymphoma (NHL), according to the FDA.
“Today marks another milestone in the development of a whole new scientific paradigm for the treatment of serious diseases. In just several decades, gene therapy has gone from being a promising concept to a practical solution to deadly and largely untreatable forms of cancer,” said FDA Commissioner Scott Gottlieb, MD.
There are approximately 72,000 new cases of NHL diagnosed in the United States annually. DLBCL is the most common NHL in adults and represents 1 in 3 new cases, according to the FDA.
Axicabtagene ciloleucel is indicated to treat adults with certain types of NHL, including DLBCL, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma and DLBCL arising from follicular lymphoma, according to the study.
The FDA cautions that the drug is not indicated to treat patients with primary central nervous system lymphoma.
“This approval demonstrates the continued momentum of this promising new area of medicine and we’re committed to supporting and helping expedite the development of these products. We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine,” Dr Gottlieb said. “That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies. We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms.”
Each treatment is customized using a patient’s own immune cells to fight cancer cells. For the therapy, patients’ T cells are collected and modified to include a gene that kills lymphoma cells, according to the release. After the T cells are altered, they are placed back in the patient.
“The approval of Yescarta brings this innovative class of CAR-T cell therapies to an additional group of cancer patients with few other options — those adults with certain types of lymphoma that have not responded to previous treatments,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research.
The safety and efficacy of the novel gene therapy was investigated in a clinical trial that included more than 100 adults with refractory or relapsed DLBCL.
The investigation found that 51% of patients treated with axicabtagene ciloleucel achieved remission, according to the release.
While this therapy may be groundbreaking, it comes with potentially lethal side effects. The FDA warns that axicabtagene ciloleucel has a boxed warning for cytokine release syndrome, which causes a high fever, flu symptoms, and neurological toxicities. Other side effects include infections, low blood cell counts, and dampened immune system.
Due to potential toxicities, the approval is subject to a risk evaluation and mitigation strategy to ensure patient safety. The FDA is also requiring hospitals to receive certification and staff to be trained how to administer the therapy and manage side effects, according to the release.
The FDA requires that patients must be informed of the potential side effects and that they should return to the hospital if they experience side effects.
Kite Pharma established a field team to provide educational and logistical training for centers that will administer the gene therapy, according to a press release. The team will provide certification to clinics to ensure the safe and effective use of axicabtagene ciloleucel. The manufacturer aims to train up to 90 centers about the drug.
“Engineered cell therapies like Yescarta represent the potential for a changing treatment paradigm for cancer patients,” said David Chang, MD, PhD, worldwide head of Research and Development and Chief Medical Officer at Kite Pharma. “Together, Gilead and Kite will accelerate studies of CAR T therapy in multiple blood cancers and advance other cell therapy approaches for solid tumors, with the goal of helping patients with diverse cancers benefit from this new era of personalized cancer therapy.”