FDA Approves Non-Hodgkin Lymphoma Gene Therapy

Gilead gains 4% after FDA approves lymphoma drug

UPDATED: FDA follows through with a groundbreaking OK for Yescarta, Gilead's new CAR-T breakthrough

The FDA has approved the CD19-directed auto T-cell therapy axicabtagene ciloleucel (axi-cel; Yescarta) for people with large B-cell lymphoma following 2 prior therapies, including for those with diffuse large B-cell lymphoma (DLBCL).

CAR-T drugs are made using a patient's own immune cells. A patient's T-cells are collected and genetically modified to include a new gene that targets and kill lymphoma cells.

The therapy, Yescarta or axicabtagene ciloleucel, will be used on adult patients with certain types of large B-cell lymphoma after other treatments fail, CNN reported on Thursday. The FDA is requiring Kite to do a long-term safety study and train hospitals to quickly spot and handle those reactions.

The novel T-cell therapy was approved after showing high levels of complete remission (CR) in a phase 2 study known as ZUMA-1. The FDA has approved a risk evaluation and mitigation strategy (REMS) for Yescarta that will inform and educate health care professionals about its associated risks.

The adverse effect profile remains the same for this treatment as for tisagenlecleucel-the drug has a boxed warning for cytokine release syndrome and for neurologic toxicities, which can both be fatal or life-threatening.

Yescarta uses gene therapy techniques called CAR-T to boost the capacity of T cells, the immune-system cells that cancer can evade.


Gilead shares stand at US$80.01 in NY. "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 Food and Drug Administration Commissioner Scott Gottlieb in a statement.

American regulators granted approval on Wednesday to a gene-altering treatment for patients suffering from lymphoma, the second-ever approval for a gene therapy procedure and one that could revolutionize how we fight cancer.

Diffuse large B-cell lymphoma is the most common type of non-Hodgkin lymphoma, representing 1-in-3 of the roughly 72,000 new cases diagnosed every year in the United States.

Michaela Almgren, a clinical assistant professor at University of South Carolina College of Pharmacy, pointed out this will not be available in just any facility due to the many challenges surrounding extraction of a patient's T-cells, adjustment of genes, and reengineering the cells in sufficient quantity. "This approval would not have been possible without the courageous commitment of patients and clinicians, as well as the ongoing dedication of Kite's employees", Kite founder Dr. Arie Belldegrun said.

Significantly, it is the first CAR-T drug approved for those patients. "We remain committed to supporting the efficient development of safe and effective treatments that leverage these new scientific platforms".

The manufacturing site has capacity to produce volumes of the autologous therapy for 4,000-5,000 patients per year, but now Yescarta has been approved Gilead said it is looking to ramp up the number of clinics available to administer the therapy. The treatment is expensive - priced at $373,000 according to Gilead.

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