Drugs

FDA Approves Novartis’ Kymriah Cancer Gene Therapy

FDA says it has approved Novartis’ Kymriah (tisagenlecleucel) as the first gene therapy available in the U.S. An agency statement says Kymriah is indicated for treating certain pediatric and young adult patients with a form of acute lymphoblastic leukemia (ALL). Specifically, it is approved in the U.S. for treating patients up to age 25 with B-cell precursor ALL that is refractory or in second or later relapse.

Kymriah is described as a genetically-modified autologous T-cell immunotherapy. Each dose is a customized treatment created using a patient’s own T-cells. The 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 that directs the T-cells to target and kill leukemia cells that have a specific antigen on the surface. Once the cells are modified, FDA says, they are infused back into the patient to kill the cancer cells.

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 reported as 83%.

The therapy labeling includes a Boxed Warning for cytokine release syndrome (CRS), a systemic response to the activation and proliferation of chimeric antigen receptor (CAR) T-cells causing high fever and flu-like symptoms, and for neurological events. There are other serious side effects.

FDA also says it has expanded the approval for Genentech’s Acterma (tocilizumab) to treat CAR T-cell induced severe or life-threatening CRS in patients two years of age or older. It reports that in clinical trials in patients treated with CAR T-cells, 69% of patients had complete resolution of CRS within two weeks following one or two Acterma doses.

Because of the risk of CRS and neurological events, Kymriah was approved with a risk evaluation and mitigation strategy that includes elements to assure safe use. The agency says it is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

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