Novartis Seeks New Indication for Gene Therapy Kymriah

Novartis recently announced the submission of a supplemental Biologics License Application (sBLA) for its recently approved gene therapy for tisagenlecleucel (Kymriah).

Novartis is seeking FDA approval for tisagenlecleucel suspension for intravenous infusion for patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are not able to receive an autologous stem cell transplant (ASCT).

Tisagenlecleucel is a single treatment that harnesses a patient’s T cells to fight cancer.

In April 2017, tisagenlecleucel received breakthrough therapy designation for r/r DLBCL. If approved, it would be the second indication for this groundbreaking gene therapy.

In August 2017, tisagenlecleucel was the first CAR-T cell therapy to receive approval, which was 5 weeks prior to its PDUFA date, according to the release. Tisagenlecleucel is indicated to treat patients up to 25 years old with B-cell precursor acute lymphoblastic leukemia that is refractory or has relapsed at least twice.

"Kymriah represents a historic breakthrough in the evolution of individualized immunotherapy and we are committed to bringing this innovation to as many patients who may benefit as possible," said Vas Narasimhan, global head of Drug Development and chief medical officer, Novartis. "The response rates we've seen in the JULIET trial show that Kymriah has the potential to transform treatment for these patients and we look forward to collaborating with the FDA to make it available to patients for this second indication."

DLBCL is the most common form of non-Hodgkin lymphoma. While up to 60% of patients maintain remission, one-third relapse. If untreated, r/r DLBCL has a life expectancy of up to 4 months, according to the release.

The new submission is based on positive findings from the phase 2 JULIET study, which enrolled patients from 27 sites across 10 countries. Novartis reported that the data from the analysis will be presented at the American Society of Hematology meeting in December 2017.

Novartis plans to submit an additional application for tisagenlecleucel in DLBCL and pediatric ALL with the European Medicines Agency, according to the release.

"The approval of tisagenlecleucel in the treatment of children and young adults with second relapse or refractory B-cell ALL was a watershed moment in the journey for researchers to develop immunocellular therapies," said Stephen Schuster, MD, director of the Lymphoma Program and Lymphoma Translational Research, University of Pennsylvania Perelman School of Medicine. "The data show this therapy could change the treatment paradigm for patients with r/r DLBCL as we've seen durable complete responses in patients who previously relapsed or were refractory to prior therapies, and this second filing is a significant step toward realizing its potential for even more patients who are currently battling fatal blood cancers."