Kymriah Approved by FDA for Treatment of Acute Lymphoblastic Leukemia

A new drug named Kymriah (tisagenlecleucel) for treatment of acute lymphoblastic leukemia in pediatric and young adult patients has been approved in the US.

The drug, approved by the FDA, is the first gene therapy treatment to be made available in the US.

Kymriah is a cell-based gene therapy that has been genetically modified and acts as an autologous T-cell immunotherapy. Each dose of the treatment is specifically customized using the individual patient’s own T-cells. The patient’s T-cells are collected and genetically modified to include a chimeric antigen receptor (CAR) that directs the T-cells to target and kill the leukemia cells that have a specific antigen (CD19) on them. Once the modification has taken place, the patient’s T-cells are infused back into the patients to kill the cancer cells.

Kymriah is approved for use in pediatric or young adult patients with B-cell acute lymphoblastic leukemia and is designed for patients whose cancer has not responded or has returned after initial treatment. The success of the drug was exemplified in the analysis of overall remission of the cancer which saw a remission rate of 83 per cent in three months of treatment.

However, whilst the drug is a promising breakthrough in the treatment of acute lymphoblastic leukemia, it does come with some severe side-effects. Because of the side-effects, Kymriah is being approved with a risk evaluation and mitigation strategy (REMS), which includes elements to assure safe use. To further evaluate the long-term safety, Novartis is also required to conduct a post-marketing observational study involving patients treated with Kymriah. Speaking on Kymriah Peter marks, MD, PHD, director of FDA’s Centre for Biologics Evaluation and Research, said:

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease. 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.”

FDA Commissioner Scott Gottlied, MDM, added:

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer. 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.”


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