Janssen Submits Marketing Authorisation Application to the European Medicines Agency Seeking Approval of BCMA CAR-T Therapy Ciltacabtagene Autoleucel (cilta-cel) for the Treatment of Relapsed and/or Refractory Multiple Myeloma
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that they have submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) seeking approval of cilta-cel, an investigational B cell maturation antigen (BCMA)-directed chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of patients with relapsed and/or refractory multiple myeloma.
The application is supported by positive results from the ongoing Phase 1b/2 CARTITUDE-1 study, investigating the safety and efficacy of cilta-cel.1,2 The latest results were presented at the American Society of Hematology (ASH) 2020 Annual Meeting. Clinical development is ongoing with patients enrolled globally in various studies, including sites in Europe, the United States of America, China and Japan.1,2
“Despite advances in the treatment of multiple myeloma, there remains a high unmet need, especially for patients whose disease continues to progress,” said Peter Lebowitz, M.D., PhD., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Through our collaboration with Legend Biotech, we continue to expedite the development of cilta-cel with a focus and priority on the patients who may benefit from this novel immunotherapy in the future.”
CAR‑T therapy is a highly personalised treatment platform where a patient’s own T-cells are re-programmed to recognise and attack cancer cells.3 In early 2021, the EMA granted accelerated assessment for cilta-cel.4 Accelerated assessment is granted when a medicinal product is expected to be of major public health interest and a therapeutic innovation, and can significantly reduce the review timelines to evaluate an MAA.5
“Janssen has been advancing the science of oncology for more than 30 years, and we see great opportunity in the area of cell therapy and through our innovative platforms,” says Mathai Mammen, M.D., Ph.D., Global Head, Janssen Research & Development, Johnson & Johnson. “We are continuing to harness our deep scientific expertise in multiple myeloma as we look to advance therapeutic options, deepen clinical responses, and drive towards improved patient outcomes.”
“Today’s submission to the EMA epitomises how we strive to make a meaningful impact in the multiple myeloma landscape through advancing innovative treatments for patients,” says Saskia De Haes, Vice President, EMEA Regulatory Affairs, Janssen R&D BE. “We look forward to working in partnership with health authorities, as part of the accelerated assessment process, to support these patients by ensuring timely access to the latest therapeutic options.”
A Biologics License Application seeking approval of cilta-cel for the treatment of relapsed and/or refractory multiple myeloma is currently under review by the United States Food and Drug Administration.6
# ENDS #
CARTITUDE-1 (NCT03548207) is an ongoing Phase 1b/2, open-label, multicentre study evaluating the safety and efficacy of JNJ-68284528 (JNJ-4528) in adults with relapsed or refractory multiple myeloma who have received at least three prior lines of therapy or are double refractory to a proteasome inhibitor (PI) and an immunomodulatory drug (IMiD); have received a PI, IMiD and an anti-CD38 antibody.2 The primary objective of the Phase 1b portion of the study is to characterise the safety and confirm the dose of JNJ-68284528 (JNJ-4528), which was informed by the first-in-human study with LCAR-B38M CAR-T cells (LEGEND-2).2 The primary objective for the Phase 2 portion of the study is to evaluate the efficacy of JNJ-4528 (primary endpoint: overall response rate as defined by the International Myeloma Working Group response criteria).2
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. Cilta-cel is a differentiated CAR-T therapy with two BCMA-targeting single domain antibodies.1 CAR-T cells are an innovative approach to targeting cancer cells by harnessing the power of a patient’s own immune system. 7 BCMA is a protein that is highly expressed on myeloma cells.8
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.9 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.2,10
In 2019, cilta-cel was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA).11 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.12 In 2020, the European Commission granted orphan designation for cilta-cel.13
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.14 In Europe, 50,918 people were diagnosed with MM in 2020, and more than 32,400 patients died.15 Around 50 percent of newly diagnosed patients do not reach five-year survival,16 and approximately 10 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.20 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.21 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.22
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 R&D BE, and Janssen Research & Development, LLC 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 ciltacabtagene autoleucel (cilta-cel). 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 materialize, actual results could vary materially from the expectations and projections of Janssen Pharmaceutica NV, 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 behaviour 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 January 3, 2021, 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 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.
2 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 April 2021.
3 Cancer Research UK. CAR-T cell Therapy. Available at: https://www.cancerresearchuk.org/about-cancer/cancer-in-general/treatment/immunotherapy/types/CAR-T-cell-therapy. Last accessed April 2021.
4 Janssen Pharmaceutica NV. 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. Available at: https://www.janssen.com/emea/sites/www_janssen_com_emea/files/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_.pdf. Last accessed April 2021
5 EMA. Accelerated Assessment. Available at: https://www.ema.europa.eu/en/human-regulatory/marketing-authorisation/accelerated-assessment. Last accessed April 2021.
6 Johnson and Johnson. 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: April 2021.
7 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
8 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.
9 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 April 2021.
10 Chen L et al., 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). Blood 2019; 134 (Supplement_1): 1858.
11 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 April 2021.
12 European Medicines Agency. PRIME Factsheet. Available at: https://www.ema.europa.eu/en/human-regulatory/research-development/prime-priority-medicines Last accessed April 2021.
13 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 April 2021.
14 American Society of Clinical Oncology. Multiple myeloma: introduction. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/introduction Last accessed: April 2021.
15 GLOBOCAN 2020. Cancer Today Population Factsheets: Europe Region. Available at: https://gco.iarc.fr/today/data/factsheets/populations/908-europe-fact-sheets.pdf. Last accessed: April 2021.
16 Cancer.Net. Multiple Myeloma: Statistics. Available at: https://www.cancer.net/cancer-types/multiple-myeloma/statistics. Last accessed: April 2021.
17 Jung SH et al., Risk factors associated with early mortality in patients with multiple myeloma who were treated upfront with a novel agents containing regimen. BMC Cancer. 2016;16:613.
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 National Cancer Institute. NCI dictionary of cancer terms: relapsed. Available at: https://www.cancer.gov/publications/dictionaries/cancer-terms?CdrID=45866. Last accessed: April 2021.
21 American Cancer Society. Multiple myeloma: early detection, diagnosis and staging. Available at: https://www.cancer.org/content/dam/CRC/PDF/Public/8740.00.pdf. Last accessed: April 2021.
22 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;26:149-57.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
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
SentinelOne Receives Highest Score for Type B Use Case in the Gartner 2021 Critical Capabilities for Endpoint Protection Platforms13.5.2021 16:00:00 CEST | Press release
SentinelOne, the autonomous cybersecurity platform company, today announced that Gartner has positioned SentinelOne with the highest score in use case Type B in Gartner’s 2021 Critical Capabilities for Endpoint Protection Platforms report1. SentinelOne received the highest score for all three customer types out of 19 vendors. Gartner says, “Type B organizations aim to stay relatively current on technology without getting too far ahead or behind their competition.” “We believe receiving the highest product score among organizations prioritizing overall value in the 2021 Critical Capabilities for Endpoint Protection Platforms validates our vision and execution in delivering an AI-powered platform purpose built for the demands of the mainstream enterprise market,” said Raj Rajamani, Chief Product Officer, SentinelOne. “These organizations represent the largest market segment out of Gartner’s use cases and prioritize technology deployments that improve their organization’s productivity, pr
Wipro Has Earned the Microsoft Windows Virtual Desktop Advanced Specialization13.5.2021 15:12:00 CEST | Press release
Wipro Limited (NYSE: WIT, BSE: 507685, NSE: WIPRO), a leading global information technology, consulting and business process services company, today said it has earned the Microsoft Windows Virtual Desktop advanced specialization, a validation of a partner’s deep knowledge, extensive experience and expertise in deploying, scaling and securing virtual desktop infrastructure on Azure. Only partners that meet stringent criteria around customer success and staff skilling, as well as pass a third-party audit of their Windows Virtual Desktop technical practices are able to earn the Microsoft Windows Virtual Desktop advanced specialization. Supporting secure remote work for employees is more critical than ever. Windows Virtual Desktop is a Microsoft solution that seamlessly integrates with other Microsoft products and allows customers to implement virtual desktops in a scalable, secure, and cost-effective way. Partners with validated capabilities in implementing Windows Virtual Desktop can he
Schlumberger Appoints Dr. Katharina Beumelburg as Chief Strategy and Sustainability Officer13.5.2021 15:00:00 CEST | Press release
Schlumberger announced today the appointment of Dr. Katharina Beumelburg to the position of Chief Strategy and Sustainability Officer, Schlumberger Limited, reporting to Olivier Le Peuch, Chief Executive Officer. The appointment is effective Monday, May 17. As a member of the executive team, Dr. Beumelburg will oversee corporate strategy, sustainability, marketing and communications activities across the Company. Dr. Beumelburg joins Schlumberger from a global technology company, Siemens, where she has held various leadership positions including strategy development incorporating sustainability; management consulting; business excellence; and operations management. In her most recent position, she led the global Transmission Services business. “I am delighted to welcome Katharina to the Schlumberger team at a pivotal time for the company, the energy industry and our planet. Sustainability is increasingly core to our performance strategy, through which we will realize our vision,” said
Ferring and Rebiotix to Present Landmark Data for Investigational Microbiota-based Live Biotherapeutic RBX2660 at Digestive Disease Week ® (DDW) 202113.5.2021 14:00:00 CEST | Press release
Ferring Pharmaceuticals and Rebiotix, a Ferring Company, today announced that Phase 3 data evaluating its investigational microbiota-based live biotherapeutic RBX2660 for reduction of recurrent Clostridioides difficile(C. difficile) infection will be presented as part of an invited talk at Digestive Disease Week® (DDW) 2021. The congress will take place virtually from May 21-23, 2021. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210513005129/en/ “These data are the pinnacle of a decade of ground-breaking research and development, driven by the goal of helping those who are experiencing the debilitating effects of recurrent C. difficile infection,” said Lee Jones, Founder, President and Chief Executive Officer of Rebiotix, a Ferring Company. “We’re striving to deliver innovative therapeutics to help people live better lives, and are proud to be pioneering a new category of scientifically-validated, microbiome-based therapeu
S&P Global Ratings Recognizes PMI’s Business Transformation as Industry Differentiator13.5.2021 13:59:00 CEST | Press release
Philip Morris International Inc. (PMI) (NYSE:PM) is transforming its business to deliver a smoke-free and sustainable future. Today, the company announced that S&P Global Ratings’ ESG Evaluation report has assessed PMI’s approach to environmental, social, and governance (ESG) topics and confirmed that PMI has positively differentiated itself within the tobacco sector. The S&P Global Ratings ESG evaluation assesses a company’s ESG strategy and ability to prepare for potential future risks and opportunities, and provides a forward-looking, long-term opinion of a company’s readiness for disruptive ESG risks and opportunities. It provides an overall score that allows comparison with other entities globally, including sector peers, and consists of a combined sector/region score, an entity-specific score, and a preparedness score. Based on entity-specific scores — designed to indicate how a company is actively and effectively managing its exposure to ESG risks and opportunities compared with
Genius Sports to Announce First Quarter 2021 Results on May 2013.5.2021 13:00:00 CEST | Press release
Genius Sports Limited (NYSE:GENI) (“Genius” or “GSL”) today announced that it will release its first quarter 2021 results before 8:00AM EDT on Thursday, May 20, 2021. At 8:00AM EDT on the same day, Genius will host a conference call to discuss the results. Genius’ earnings press release and related materials will be available at investors.geniussports.com. To listen to the live audio webcast and Q&A, please visit Genius’ investor relations website at investors.geniussports.com. A replay of the webcast will be available on the website within 24 hours after the call. About Genius Sports Genius Sports is the official data, technology and commercial partner that powers the global ecosystem connecting sports, betting and media. We are a global leader in digital sports content, technology and integrity services. Our technology is used in over 150 countries worldwide, empowering sports to capture, manage and distribute their live data and video, driving their digital transformation and enhanc
Knopp Biosciences to Present Details of Positive Results From the Phase 2 EXHALE Trial of Oral Dexpramipexole in Moderate-to-Severe Eosinophilic Asthma at ATS 202113.5.2021 12:00:00 CEST | Press release
Knopp Biosciences LLC today announced it will present detailed results of its positive Phase 2 dose-ranging EXHALE trial of the novel oral eosinophil-lowering drug dexpramipexole in patients with moderate-to-severe asthma in a virtual poster session at the ATS 2021 International Conference. “We look forward to sharing comprehensive results of the EXHALE trial, which underscore the readiness of dexpramipexole for further development in eosinophilic asthma,” said Michael Bozik, M.D., president and CEO of Knopp. “Monoclonal antibody treatments approved for asthma demonstrate that reduction in eosinophils is associated with significantly improved outcomes, but the need remains for an oral alternative in the eosinophilic asthma treatment paradigm.” Following are details of the poster presentation: Poster Title Abstract Number / Presentation Details Oral Dexpramipexole Efficacy in Lowering Blood Eosinophils in Patients with Moderate to Severe Uncontrolled Eosinophilic Asthma A1359 Poster Pre