Kymriah filed at FDA to treatrelapsed or refractory diffuse large B-cell lymphoma.- Novartis.

Novartis has announced that the company has submitted a supplemental Biologics License Application (sBLA) to the FDA for Kymriah (tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem cell transplant .

In April 2017, Novartis received Breakthrough Therapy designation for r/r DLBCL which, if approved, would be the second indication for Kymriah. In August 2017, Kymriah became the first available chimeric antigen receptor T cell (CAR-T) therapy when it received FDA approval five weeks prior to its PDUFA date and was launched for patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or has relapsed at least twice. Kymriah is a novel immunocellular therapy and a one-time treatment that uses a patient's own T cells to fight cancer.

The submission is based on results from the Novartis-sponsored, global, multi-center Phase II JULIET study (NCT02445248), the first global, multi-center registration study for Kymriah in adult patients with r/r DLBCL. JULIET was conducted in collaboration with Penn and is the largest study examining a CAR-T therapy exclusively in DLBCL, enrolling patients from 27 sites in 10 countries across the US, Canada, Europe, Australia and Japan. Data from the six-month primary analysis of JULIET will be presented at the annual meeting of the American Society of Hematology (ASH) in December 2017.

Novartis plans to submit an additional application for marketing authorization for Kymriah with the European Medicines Agency in both DLBCL and pediatric ALL later this year. Novartis plans additional regulatory filings for Kymriah outside the US and EU in 2018.

Comment: DLBCL is the most common form of non-Hodgkin lymphoma (NHL), a cancer of the lymphatic system, accounting for up to 40% of all NHL cases globally. Roughly 50-60% of patients with DLBCL achieve and maintain complete remission after first-line therapy; however, roughly one-third of patients relapse after receiving first-line treatment. Only about 25% of patients with r/r DLBCL are eligible for autologous stem cell transplant, the mainstay of secondary treatment. If left untreated, r/r DLBCL has a life expectancy of three to four months.