Drugs

Kite Pharma CAR-T Cell Therapy Approved

FDA has approved a Kite Pharma NDA for Yescarta (axicabtagene ciloleucel), a cell-based gene therapy for treating adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment. The chimeric antigen receptor (CAR) T cell therapy is the second gene therapy approved and the first for certain types of non-Hodgkin lymphoma (NHL), an agency release says. In August, Novartis’ Kymriah (tisagenlecleucel) gained approval as the first gene therapy available in the U.S. (see story).

“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,” commissioner Scott Gottlieb is quoted in the release as saying. “We will soon release a comprehensive policy to address how we plan to support the development of cell-based regenerative medicine. That policy will also clarify how we will apply our expedited programs to breakthrough products that use CAR-T cells and other gene therapies.”

Yescarta is a customized treatment developed using a patient’s own immune system to help fight the lymphoma. The patient’s T-cells are collected and genetically modified to include a new gene that targets and kills the lymphoma cells, the agency says. They are then infused back into the patient. Approval was based on data from more than 100 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after therapy was 51%, it says.

The approval also 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 neurologic toxicities. “Other side effects include serious infections, low blood cell counts and a weakened immune system,” FDA says.

The FDA granted Yescarta Priority Review and Breakthrough Therapy designations. Yescarta also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The Yescarta application was reviewed using a coordinated, cross-agency approach. The clinical review was conducted by FDA's Oncology Center of Excellence, while CBER conducted all other aspects of review and made the final product approval determination.

Gupta Raises Concerns About Overton’s Independence, Ideology

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Overton Confirmation Remains Uncertain After Senate Hearing

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FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome