FDA Accepts sBLA, Grants Priority Review for Tisagenlecleucel in Adults With DLBCL

Today, the U.S. Food and Drug Administration (FDA) accepted for Priority Review a supplemental Biologics License Application (sBLA) for tisagenlecleucel (Kymriah) suspension for intravenous infusion (formerly CTL019) for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) who are ineligible for or relapse after autologous stem cell transplant (ASCT). In addition, the European Medicines Agency (EMA) has granted accelerated assessment to the Marketing Authorization Application (MAA) for tisagenlecleucel for the treatment of children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (ALL) and for adult patients with relapsed or refractory DLBCL who are ineligible for ASCT. If approved by the FDA and EMA, tisagenlecleucel would represent the first chimeric antigen receptor (CAR) T-cell therapy available for two distinct indications in non-Hodgkin lymphoma and B-cell ALL.  

Tisagenlecleucel became the first CAR T-cell therapy to receive regulatory approval when it was approved by the FDA in August 2017 for the treatment of patients up to 25 years of age with B-cell precursor ALL that is refractory or in second or later relapse.

“The first approval of a CAR T-cell 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. “The Priority Review designation and accelerated assessment signal that the FDA and EMA have recognized the potential of tisagenlecleucel 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.”

Tisagenlecleucel is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer. Tisagenlecleucel uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence.

JULIET and ELIANA Trials

The regulatory applications in the United States and European Union are based on data from global clinical trial program of tisagenlecleucel in children and young adults with relapsed or refractory B-cell ALL and adult patients with relapsed or refractory DLBCL demonstrating the efficacy and safety of tisagenlecleucel across studies. Results from the phase II JULIET trial served as the basis of the sBLA and MAA for tisagenlecleucel in adult patients with relapsed or refractory DLBCL. Results from the phase II ELIANA study were submitted as part of the MAA for tisagenlecleucel in children and young adults with relapsed or refractory B-cell ALL.

JULIET is the first multicenter global registration study for tisagenlecleucel in adult patients with relapsed or refractory DLBCL. JULIET is the largest study examining a CAR T-cell therapy in DLBCL, enrolling patients from 27 sites in 10 countries across the United States, Canada, Australia, Japan, and Europe, including Austria, France, Germany, Italy, Norway, and the Netherlands. Data from the 6-month primary analysis of JULIET were presented at the 59th American Society of Hematology Annual Meeting & Exposition (Abstract 577).

ELIANA is the first pediatric global CAR T-cell therapy registration trial, examining patients in 25 centers in the United States, Canada, Australia, Japan, and Europe, including Austria, Belgium, France, Germany, Italy, Norway, and Spain.

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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