Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough    Therapy designation for treatment of adult patients with r/r    DLBCL.    
    Novartis announced that the U.S. Food and Drug Administration    (FDA) has granted Breakthrough Therapy designation to CTL019,    an investigational chimeric antigen receptor T cell (CAR-T)    therapy, for the treatment of adult patients with relapsed and    refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who    have failed two or more prior therapies.  
    This is the second indication for which CTL019 has received    this designation; the first being for the treatment of r/r    B-cell acute lymphoblastic leukemia (ALL) in pediatric and    young adult patients.  
    "At Novartis, we are eager to unlock the full potential of    CTL019, including the potential to help patients with r/r    DLBCL," said Vas Narasimhan, Global Head of Drug Development    and Chief Medical Officer, Novartis. "We look forward to    working closely with the FDA to help bring this potential new    treatment option to patients as soon as possible."  
    CAR-T cell therapy is different from typical small molecule or    biologic therapies currently on the market because it is    manufactured for each individual patient. During the treatment    process, T cells are drawn from a patient's blood and    reprogrammed in the laboratory to create T cells that are    genetically coded to hunt the patient's cancer cells and other    B-cells expressing a particular antigen.  
    CTL019 was first developed by the University of Pennsylvania    (Penn). In 2012, Novartis and Penn entered into a global    collaboration to further research, develop and then    commercialize CAR-T cell therapies, including CTL019, for the    investigational treatment of cancers. Through the    collaboration, Novartis holds the worldwide rights to CARs    developed with Penn for all cancer indications.  
    In March 2017, Novartis announced that the FDA accepted the    company's Biologics License Application filing and granted    priority review for CTL019 in the treatment of r/r pediatric    and young adult patients with B-cell ALL.  
    The Breakthrough Therapy designation is based on data from the    multi-center phase II JULIET study (NCT02445248), which is    evaluating the efficacy and safety of CTL019 in adult patients    with r/r DLBCL. JULIET is the second global CAR-T trial,    following the Novartis ELIANA study (NCT02435849) investigating    CTL019 in r/r B-cell ALL. Findings from JULIET are expected to    be presented at an upcoming medical congress.  
    "We are encouraged by the FDA's recognition in the potential of    CTL019 for this indication, which follows our promising studies    of this therapy for ALL and the FDA filing by Novartis in    pediatric and young adult ALL that received priority review,"    said the Penn team's leader, Carl June, M.D., director of the    Center for Cellular Immunotherapies in the Perelman School of    Medicine at the University of Pennsylvania. "Work with our    collaborators at trial sites across the world is paving a path    to bring personalized cell therapies to more patients with    these devastating blood cancers."  
    According to FDA guidelines, treatments that receive    Breakthrough Therapy designation are those that treat a serious    or life threatening disease or condition and demonstrate a    substantial improvement over existing therapies on one or more    clinically significant endpoints based on preliminary clinical    evidence. The designation also indicates that the agency will    expedite the development and review of CTL019 in adults with    r/r DLBCL.  
    This marks the 14thBreakthrough Therapy    designation for Novartis since the FDA initiated the program in    2013, underscoring an emphasis to develop innovative treatments    in disease areas with significant unmet need.  
    DLBCL is the most common form of lymphoma and accounts for    approximately 30 percent of all non-Hodgkin lymphoma    cases1. Ten to 15 percent of DLBCL patients fail to    respond to initial therapy or relapse within three months of    treatment, and an additional 20 to 25 percent relapse after    initial response to therapy2.  
    Because CTL019 is an investigational therapy, the safety and    efficacy profile has not yet been established. Access to    investigational therapies is available only through carefully    controlled and monitored clinical trials. These trials are    designed to better understand the potential benefits and risks    of the therapy. Because of the uncertainty of clinical trials,    there is no guarantee that CTL019 will ever be commercially    available anywhere in the world.    ____________________________________________________  
    References:  
    1 American Society of Clinical Oncology. Lymphoma -    Non-Hodgkin: Subtypes (Dec. 2016 revision).http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes.    Accessed March 2017.  
    2 Sehn, L. Paramount prognostic factors that guide    therapeutic strategies in diffuse large B-cell lymphoma.    Hematology, December 2012; 1; 402-409.  
    (Source: GlobeNewswire)  
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CAR-T Cell Therapy Receives FDA Breakthrough Designation - Pharmaceutical Processing