On February 1, 2021 The Janssen Pharmaceutical Companies of Johnson & Johnson reported 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) (Press release, Janssen Pharmaceuticals, FEB 1, 2021, View Source [SID1234574470]). 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

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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) (Free ASH Whitepaper) 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

About CARTITUDE-1
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