On December 20, 2016 Juno Therapeutics, Inc. (NASDAQ: JUNO) and Celgene Corporation (NASDAQ: CELG), reported that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to investigational drug JCAR017 for the treatment of patients with relapsed/refractory (r/r) aggressive large B-cell non-Hodgkin lymphoma (NHL), including diffuse large B-cell lymphoma (DLBCL), not otherwise specified (de novo or transformed from indolent lymphoma), Primary Mediastinal B-cell Lymphoma (PMBCL) or Grade 3B Follicular Lymphoma (Press release, Celgene Schedule your 30 min Free 1stOncology Demo! JCAR017 uses a defined CD4:CD8 cell composition and 4-1BB as the costimulatory domain, which differentiates it from other CD19-directed CAR T product candidates in clinical development. Earlier this month, data from multiple phase I studies with JCAR017 in non-Hodgkin lymphoma and pediatric acute lymphoblastic leukemia were presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.
, DEC 20, 2016, View Source [SID1234517136]). In addition, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) and Committee for Advanced Therapies (CAT) have granted JCAR017 access to the Priority Medicines (PRIME) scheme for r/r DLBCL.
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"The Breakthrough Therapy designation from the FDA and PRIME eligibility from EMA for JCAR017 highlight the need for new treatment options for patients with DLBCL, particularly for the significant number of patients who do not respond to initial therapy or with relapsed disease," said Mark J. Gilbert, M.D., Juno’s Chief Medical Officer. "Early data with JCAR017 in a range of B-cell malignancies has been encouraging and we look forward to initiating a pivotal trial in patients with relapsed or refractory DLBCL in the U.S. in 2017."
"The encouraging news of the FDA Breakthrough Therapy and EMA PRIME designations reflect the agencies’ emphasis on accelerating the development and assessment of potential new options for serious blood cancers like DLBCL," said Jay Backstrom, M.D., Chief Medical Officer and Head of Global Regulatory Affairs for Celgene. "We will continue to work closely with our partners at Juno and with the agencies to move the JCAR017 program forward in the interest of patients with relapsed or refractory DLBCL."
The Breakthrough Therapy designation and PRIME eligibility were granted by the FDA and EMA, respectively, on the basis of early clinical results with JCAR017 in r/r DLBCL. According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of medicines with early evidence of potential clinical benefit in serious diseases. PRIME was launched by the EMA earlier this year and enables accelerated assessment of therapeutic applications that target an unmet medical need, focusing on medicines that may offer a major therapeutic advantage over existing treatments, or benefit patients with no treatment options.
JCAR017 is not approved in any country. FDA’s Breakthrough Therapy designation and access to EMA’s Priority Medicines scheme does not constitute or guarantee a future approval.