FDA Gives Priority Review to Kymriah for Second Rare Cancer Indication

In August, the U.S. Food and Drug Administration (FDA) approved a CAR-T cell therapy for the first time when it gave Novartis the go-ahead for Kymriah (tisagenlecleucel), intended for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is either refractory or in second or later relapse.

This morning, Novartis was granted U.S. FDA Priority Review for the drug in patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT).

Kymriah is a one-time autologous T-cell immunotherapy treatment. Each dose of it is a customized treatment created using an individual patient’s own T-cells. The T-cells are collected and sent to a manufacturing center where they are altered to include a new gene that contains a chimeric antigen receptor (CAR). The CAR will direct the T-cells to target and eliminate leukemia cells that have CD19, a specific antigen, on the surface.

Once modified, the cells are infused back into the patient to kill the cancer cells.

"The first approval of a CAR-T therapy truly redefined the future of the cancer treatment landscape, and we are only at the beginning of this new era in cancer care," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development in a press release. "The Priority Review designation and accelerated assessment signal that the FDA and EMA have recognized the potential of Kymriah to provide a much-needed therapeutic option for these patients with relapsed or refractory B-cell ALL and DLBCL. We are now focused on working with these regulatory agencies to bring this potentially transformative therapy to more patients."

The regulatory applications in the U.S. and European Union (EU) are based on data from the Novartis-sponsored global clinical trial program of Kymriah, demonstrating the efficacy and safety of the drug in children and young adults in the indication, specifically the pivotal phase 2 JULIET clinical trial. The study enrolled patients from 27 sites in 10 countries across the U.S., Canada, Australia, Japan, and Europe.

At the 6-month mark of the study, 30% of patients treated with Kymriah were in complete response, with a 74% relapse-free rate after onset of response.

Novartis has plans for additional regulatory submissions for Kymriah in pediatric and young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL beyond the U.S. and EU in 2018.

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