Janssen Announces CAR-T Therapy Ciltacabtagene Autoleucel (Cilta-cel) Accepted for Accelerated Assessment in Europe for the Treatment of Patients with Heavily Pretreated Multiple Myeloma
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) will perform an accelerated assessment of the Marketing Authorisation Application (MAA) for the B-cell maturation antigen (BCMA)-targeted chimeric antigen receptor T-cell (CAR-T) therapy ciltacabtagene autoleucel (cilta-cel). Accelerated assessment is granted by the CHMP when a medicinal product is expected to be of major public health interest and therapeutic innovation and can significantly reduce the review timelines to evaluate an MAA.1
Cilta-cel is an investigational BCMA-directed CAR-T therapy in development for the treatment of adults with relapsed and/or refractory multiple myeloma.2 CAR‑T therapy is a highly personalised technology where a patient’s own T-cells are re-programmed to target and eradicate cancer.3
“Multiple myeloma is a rare, incurable cancer, and has long been an area of focus for Janssen,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research Development, Janssen Research & Development, LLC. “We are deeply committed to improving outcomes for patients with multiple myeloma, with a goal of delivering innovations that have the potential to expand current remission periods and improve quality of life.”
The cilta-cel MAA, which is targeted for submission in the first half of 2021, is supported by the positive results from the Phase 1b/2 CARTITUDE-1 study.2 The latest results from the CARTITUDE-1 study were presented at the American Society of Hematology (ASH) 2020 Annual Meeting.2
“We are excited that the potential clinical benefit of cilta-cel is being recognised and now look forward to working with the EMA to bring this highly innovative treatment to patients in need,” said Saskia De Haes, Vice President, EMEA Regulatory Affairs, Janssen R&D BE.
This accelerated approval milestone in Europe follows the December 2020 announcement of a rolling submission of the Biologics License Application (BLA) for cilta-cel to the U.S. Food and Drug Administration (FDA).4
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of cilta-cel in adults with relapsed and/or refractory multiple myeloma, 99 percent of whom were refractory to the last line of treatment; 88 percent of whom were triple-class refractory, meaning their cancer did not, or no longer responds to an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody.2,5
The primary objective of the CARTITUDE-1 study is to characterise the safety and confirm the dose of cilta-cel, informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2) and to evaluate the efficacy of cilta-cel with overall response as the primary endpoint.2,5
About Ciltacabtagene Autoleucel (cilta-cel)
Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy for the treatment of patients with multiple myeloma. The design comprises a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies.2 CAR-T cells are an innovative approach to eradicating cancer cells by harnessing the power of a patient’s own immune system.6 BCMA is a protein that is highly expressed on myeloma cells.7
In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech to develop and commercialise cilta-cel.8 In May 2018, Janssen initiated a Phase 1b/2 CARTITUDE-1 trial (NCT03548207) to evaluate the efficacy and safety of cilta-cel in adults with relapsed and/or refractory multiple myeloma, informed by the LEGEND-2 study results.2,9
In April 2019, cilta-cel was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA).10 PRIME offers enhanced interaction and early dialogue with developers of promising medicines, to optimise drug development plans and speed up evaluation of cutting-edge, scientific advances that target a high unmet medical need.11 In February 2020, the European Commission granted orphan designation for cilta-cel.12
About Multiple Myeloma
Multiple myeloma (MM) is an incurable blood cancer that starts in the bone marrow and is characterised by an excessive proliferation of plasma cells.13 In Europe, more than 48,200 people were diagnosed with MM in 2018, and more than 30,800 patients died.14 Around 50 percent of newly diagnosed patients do not reach five-year survival,15,16 and almost 29 percent of patients with multiple myeloma will die within one year of diagnosis.17
Although treatment may result in remission, unfortunately, patients will most likely relapse as there is currently no cure.18 Refractory MM is when a patient’s disease progresses within 60 days of their last therapy.19 Relapsed cancer is when the disease has returned after a period of initial, partial or complete remission.15 While some patients with MM have no symptoms at all, others are diagnosed due to symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.20 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and require new therapies for continued disease control.21
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.
Learn more at www.janssen.com/emea. Follow us at www.twitter.com/janssenEMEA for our latest news. Janssen Pharmaceutica NV, Janssen Research & Development, LLC, Janssen R&D BE, and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
# # #
Cautions Concerning Forward-Looking Statements
This press release contains "forward-looking statements" as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of ciltacabtagene autoleucel. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Janssen Pharmaceutica NV and/or any of the other Janssen Pharmaceutical Companies and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's Annual Report on Form 10-K for the fiscal year ended December 29, 2019, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in the company’s most recently filed Quarterly Report on Form 10-Q, and the company’s subsequent filings with the Securities and Exchange Commission. Copies of these filings are available online at www.sec.gov, www.jnj.com or on request from Johnson & Johnson. None of the Janssen Pharmaceutical Companies nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.
1 EMA. Accelerated Assessment. Available at: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/accelerated-assessment. Last accessed January 2021.
2 Madduri, D et al. Cartitude-1: Phase 1b/2 Study of Ciltacabtagene Autoleucel, a B-Cell Maturation Antigen–Directed Chimeric Antigen Receptor T Cell Therapy, in Relapsed/Refractory Multiple Myeloma. Oral Presentation. Presented at 2020 American Society of Hematology Annual Meeting.
3 NHS. CAR-T Therapy. Available at: https://www.england.nhs.uk/cancer/cdf/car-t-therapy/. Last accessed January 2021.
4 JnJ.com. Janssen Initiates Rolling Submission of a Biologics License Application to U.S. FDA for BCMA CAR-T Therapy Ciltacabtagene Autoleucel (cilta-cel) for the Treatment of Relapsed and/or Refractory Multiple Myeloma. Available at: https://www.jnj.com/janssen-initiates-rolling-submission-of-a-biologics-license-application-to-u-s-fda-for-bcma-car-t-therapy-ciltacabtagene-autoleucel-cilta-cel-for-the-treatment-of-relapsed-and-or-refractory-multiple-myeloma. Last accessed January 2021.
5 ClinicalTrials.gov. A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-Cell Maturation Antigen (BCMA) in Participants With Relapsed or Refractory Multiple Myeloma (CARTITUDE-1). Available at: https://clinicaltrials.gov/ct2/show/NCT03548207 Last accessed January 2021.
6 Hay A, Cheung M. CAR T-cells: costs, comparisons, and commentary. J Med Econ. 2019: 22(7): 613-615, DOI: 10.1080/13696998.2019.1582059
7 Cho SF, Anderson KC, Tai YT. Targeting B-cell maturation antigen (BCMA) in multiple myeloma: potential uses of BCMA-based immunotherapy. Front Immunol. 2018;9:18-21.
8 JnJ.com Janssen Enters Worldwide Collaboration and License Agreement with Chinese Company Legend Biotech to Develop Investigational CAR-T Anti-Cancer Therapy. Available at: https://www.jnj.com/media-center/press-releases/janssen-enters-worldwide-collaboration-and-license-agreement-with-chinese-company-legend-biotech-to-develop-investigational-car-t-anti-cancer-therapy Last accessed January 2021.
9 Chen L, Xu J, Fu Sr W, Jin S, Yang S, Yan S, Wu W, Liu Y, Zhang W, Weng X, Wang Y. Updated phase 1 results of a first-in-human open-label study of Lcar-B38M, a structurally differentiated chimeric antigen receptor T (CAR-T) cell therapy targeting B-cell maturation antigen (Bcma).
10 JnJ.com. Janssen Announces Investigational CAR-T Therapy JNJ-68284528 Granted PRIME Designation by the European Medicines Agency. Available at: https://www.jnj.com/janssen-announces-investigational-car-t-therapy-jnj-68284528-granted-prime-designation-by-the-european-medicines-agency Last accessed January 2021.
11 European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines Last accessed January 2021.
12 European Medicines Agency (EMA). Public summary of opinion on orphan designation Available at: https://www.ema.europa.eu/en/documents/orphan-designation/eu/3/20/2252-public-summary-positive-opinion-orphan-designation-autologous-human-t-cells-genetically_en.pdf. Last accessed January 2021.
13 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed January 2021.
14 GLOBOCAN 2018. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf. Last accessed January 2021.
15 American Society of Clinical Oncology. Multiple Myeloma: Statistics. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/statistics. Last accessed January 2021.
16 Cancer Research UK. Myeloma Statistics. Available at: https://www.cancerresearchuk.org/health-professional/cancer-statistics/statistics-by-cancer-type/myeloma#heading-Two Last accessed January 2021.
17 Costa LJ, Gonsalves WI, Kumar SK. Early mortality in multiple myeloma. Leukemia. 2015;29:16168.
18 Abdi J, Chen G, Chang H, et al. Drug resistance in multiple myeloma: latest findings and new concepts on molecular mechanisms. Oncotarget. 2013;4:2186–2207.
19 Richardson P, Mitsiades C, Schlossman R, et al. The treatment of relapsed and refractory multiple myeloma. Hematology Am Soc Hematol Educ Program. 2007:317-23.
20 American Cancer Society. Multiple Myeloma: Symptoms and Signs. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/symptoms-and-signs. Last accessed January 2021.
21 Kumar SK, Lee JH, Lahuerta JJ, et al., Risk of progression and survival in multiple myeloma relapsing after therapy with IMiDs and bortezomib: a multicenter international myeloma working group study. Leukemia. 2012;2621:149-57.
Mobile: +31 621-385-718
Office: +1 732-524-2955
Office: +1 732-524-3922
About Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
CGTN: China Outlines Plan to Address Economic Challenges in Next 5 Years5.3.2021 14:02:00 CET | Press release
China has set a gross domestic product (GDP) growth target of over six percent for 2021 and outlined a plan to promote innovation, green development, and common prosperity in the next five years. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210305005288/en/ Addressing the country's annual legislative session, Premier Li Keqiang on Friday explained how China is going to tackle the numerous challenges it faces in a new development stage. "China remains in an important period of strategic opportunity for development. Yet, there are changes in both the opportunities and challenges we face," Li said when delivering a government work report at the opening meeting of the fourth session of the 13th National People's Congress (NPC), China's top legislature. 'Extraordinary year' Describing 2020 as "an extraordinary year in the history of the People's Republic of China," the premier underscored China's "major strategic success" in fi
Everbridge Awarded Revolutionary New Public Warning Patent for Technology that Automates the Selection of the Optimal Communications Channels to Reach the Broadest, Hyper-Targeted Populations – as Fast as Possible – During a Crisis5.3.2021 13:30:00 CET | Press release
Everbridge, Inc. (NASDAQ: EVBG), the global leader in critical event management (CEM), today announced the company received a revolutionary new patent related to its world-class Public Warning system. The patent pertains to technology focused on hybrid population alerting systems and intelligent sending of messages in public mobile networks. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210305005260/en/ Everbridge Awarded Revolutionary New Public Warning Patent (Photo: Business Wire) Everbridge represents the first population alerting provider to serve the entire populations of 11 countries in Europe, Asia, Oceania, the Middle East, Africa, and the Americas, supporting five European Union (EU) countries in conjunction with the EU mandate requiring member countries to have a population-wide alerting system in place by June 2022. In compliance with GDPR, Everbridge Public Warning neither collects nor stores personally identif
COVID-19’s Economic Pinch Drives More Nordic Enterprises Toward Business Process Automation for Competitive Edge5.3.2021 10:00:00 CET | Press release
Large enterprises in the Nordics have been implementing business process automation over the last three years, and more organizations now see the urgency of adopting these technologies to stay competitive as COVID-19 strains the regional economy, according to a new report published today by Information Services Group (ISG) (Nasdaq: III), a leading global technology research and advisory firm. The 2020 ISG Provider Lens™Intelligent Automation – Solutions and Services report for the Nordics finds banking, financial services and insurance companies have led the region in adopting business process automation, seeking higher productivity, cost savings and improved customer experience. Manufacturing, retail and other sectors are also embracing the technologies, which include conversational AI, natural-language processing and AI for IT operations (AIOps). “Early adopters of automation in the Nordics are stepping up digital transformation to gain an advantage when they need it most,” said Jan
International Fashion Designer Anna Chybisova Draws Major Investments Worth $9 Million for her Brand Maison d’Angelann5.3.2021 09:52:00 CET | Press release
Following a series of successful market takeovers, Anna Chybisova takes another step towards success by securing another major investment for Maison D’angelann. The Gate Business Service, a private family office based out of the United Arab Emirates, has been announced as the latest investor to back Anna’s brand globally. After having invested $7 million late last year, Gate Business Service secures an additional $2 million worth of investment against a majority stake in Maison d’Angelann fashion brand. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210305005133/en/ Anna Chybisova, Fashion Designer & Owner, Maison d’Angelann (Photo: AETOSWire) Anna Chybisova’s designs and business acumen are the foremost reasons behind such heavy investments by the new partner. Reporting sales over $2 million within UAE alone and a sold-out Spring/Summer 2021 collection has strengthened the investor confidence. This growing success and custo
Xlife Sciences AG (XLS): Joint Venture With anfass Life Technologies5.3.2021 08:48:00 CET | Press release
Xlife Sciences AG and Solothurn-based anfass Life Technologies AG have entered a joint venture. Hence, the jointly founded Quadira Biosciences AG has access to the 3D CoSeedis(TM) technology platform of abc biopply ag. This unique 3D cell technology enables the replication of human tissue for reliable testing and characterization of antibodies without animal testing. Xlife's technology platform for the development of antibodies will be used even more efficiently. As of today, Xlife Sciences AG has already identified 30 compounds with blockbuster potential with its antibody screening platform. These therapeutic antibodies have already shown to be safe and effective in humans. Xlife can modify these antibodies with utmost accuracy and increase the quality of the active ingredient. These are mainly advanced, highly potent antibodies with higher cytotoxic activity and improved side effect profile for cancer therapy. The target market potential of the pre-selected antibodies is in the range
Moody’s Analytics Wins IFRS 9 Solution Provider of the Year5.3.2021 08:30:00 CET | Press release
Moody’s Analytics has won IFRS 9 Solution Provider of the Year in the 2021 Insurance Asset Risk Awards. It is the latest recognition for our capabilities in this space following wins for IFRS 9 – Enterprise Solution of the Year and IFRS 9 – ECL Modelling Solution of the Year at last year’s Risk Technology Awards. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210304006204/en/ Central to our offering for insurers is the ImpairmentStudio™ for IFRS 9 solution, a cloud-based platform that brings together our award-winning economic scenarios, data, models, and enterprise software. Financial institutions use it to automate the credit loss impairment calculations mandated by the new IFRS 9 accounting standard. For insurers, these new financial reporting requirements call for much more granular data than was previously needed. The Moody’s Analytics offering helps them access and use all of the required data while managing and monito
Mitsubishi Heavy Industries Engineering to Test Carbon Capture Technology at Technology Centre Mongstad in Norway5.3.2021 07:43:00 CET | Press release
Mitsubishi Heavy Industries Engineering (MHIENG), part of Mitsubishi Heavy Industries (MHI) Group, has entered into an agreement with Technology Centre Mongstad (TCM) to test its proprietary solvent for capturing CO2 at the amine plant located in Mongstad, Norway. The test campaign will start in May. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210304006210/en/ Technology Centre Mongstad located at the west coast of Norway (Photo: Business Wire) The proprietary solvent to be tested is the KS-21TM, an amine-based adsorbent used in the “Advanced KM CDR ProcessTM” newly developed by MHIENG in collaboration with Kansai Electric Power Co., Inc. (KEPCO). Its long-term usage will be demonstrated in Norway, one of the world’s most advanced countries with respect to environmental regulations on CO2 capture, in a quest to achieve commercialization within 2021. Compared to the earlier KS-1TM solvent, which has been adopted at 13 comm