Rare Disease Report

FDA Approves First Gene Therapy

AUGUST 30, 2017
Mathew Shanley
History was made this morning when the U.S. Food and Drug Administration (FDA) approved the gene therapy, Kymriah (tisagenlecleucel), developed by Novartis.

The cell-based gene therapy is sanctioned in the United States 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.

B-cell precursor ALL is an assertive cancer type in which a surplus of B-cell lymphocytes is produced. It progresses quickly, and is the most common childhood cancer in the United States, affecting an estimated 3,100 patients aged 20 years and younger annually.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb, M.D. in a press release.

“New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses. At the FDA, we’re committed to helping expedite the development and review of groundbreaking treatments that have the potential to be life-saving.”

Kymriah, an autologous T-cell immunotherapy, is genetically-modified and 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 genetically modified 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.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research (CBER). “Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials.”

In one multicenter clinical trial of 63 pediatric and young adult patients with relaped or refractor C-cell precursor ALL, the safety and efficacy of Kymriah were confirmed as the overall remission rate was 83% within 3 months of treatment.

The approval has sent shock waves throughout the cancer community. American Society of Hematology (ASH) President Kenneth C. Anderson, MD, stated:

"The approval of CAR T-cell therapy for pediatric leukemia marks an important shift in the blood cancer treatment paradigm. We now have proof that it is possible to eradicate cancer by harnessing the power of a patient's own immune system. This is a potentially curative therapy in patients whose leukemia is unresponsive to other treatments and represents the latest milestone in the shift away from chemotherapy toward precision medicine."

Kymriah comes with a boxed warning for cytokine release syndrome (CRS) which causes high fever and flu-like symptoms, as well as other severe side effects including serious infections, low blood pressure, acute kidney injury, fever, and decreased oxygen.

To further assess the long-term safety, Novartis is also obligated to conduct a post-marketing observational study involving patients treated with Kymriah.

For the latest FDA applications, designations and approvals, follow Rare Disease Report on Facebook and Twitter. For breaking news delivered directly to your inbox, sign up for the RDR e-newsletter.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.