Novartis said its supplemental Biologics License Application for Kymriah (tisagenlecleucel) suspension for intravenous infusion, formerly CTL019, for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma who are ineligible for or relapse after autologous stem cell transplant (ASCT) has been accepted by FDA for Priority Review.
In addition, the European Medicines Agency has granted accelerated assessment to the Marketing Authorization Application for Kymriah for the treatment of children and young adults with r/r B-cell acute lymphoblastic leukemia and for adult patients with r/r DLBCL who are ineligible for ASCT.
Kymriah 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.
Kymriah uses the 4-1BB costimulatory domain in its chimeric antigen receptor to enhance cellular expansion and persistence. In 2012, Novartis and the University of Pennsylvania entered into a global collaboration to further research, develop and commercialize CAR-T cell therapies, including Kymriah, for the investigational treatment of cancers.
The regulatory applications in the US and EU are based on data from the Novartis-sponsored global clinical trial program of Kymriah in children and young adults with r/r B-cell ALL and adult patients with r/r DLBCL demonstrating the efficacy and safety of Kymriah across studies. Results from the pivotal phase II JULIET clinical trial served as the basis of the sBLA and MAA (applications submitted by pharmaceutical companies to health authorities when seeking approval of a new product) for Kymriah in adult patients with r/r DLCBL. Results from the pivotal phase II ELIANA study were submitted as part of the MAA for Kymriah in children and young adults with r/r B-cell ALL.
JULIET is the first multi-center global registration study for Kymriah in adult patients with r/r DLBCL. JULIET is the largest study examining a CAR-T therapy in DLBCL, enrolling patients from 27 sites in 10 countries across the US, Canada, Australia, Japan and Europe, including: Austria, France, Germany, Italy, Norway and the Netherlands. Data from the six-month primary analysis of JULIET were presented at the annual meeting of the American Society of Hematology (ASH) in December 2017.
ELIANA is the first pediatric global CAR-T cell therapy registration trial, examining patients in 25 centers in the US, Canada, Australia, Japan, and the EU, including: Austria, Belgium, France, Germany, Italy Norway, and Spain.
