CARVYKTI® (Ciltacabtagene Autoleucel), Janssen’s First Cell Therapy, for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma

In the pivotal clinical study CARTITUDE-1, 98 percent of patients with relapsed or refractory multiple myeloma responded to a one-time treatment with ciltacabtagene autoleucel and 80 percent of patients who responded experienced a stringent complete response 

The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the European Commission (EC) granted conditional marketing authorisation of CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) for the treatment of adults with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI) and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy. In December 2017, Janssen Biotech, Inc. (Janssen) entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialise cilta-cel.[1]

“Patients who have relapsed, or for whom treatment regimens have stopped working after experiencing the three major drug classes, typically face poor survival. Despite recent innovation, new therapeutic approaches are still needed,” said Maria-Victoria Mateos, M.D., Ph.D., Consultant Physician in Haematology, University Hospital of Salamanca.* “The CARTITUDE-1 data, on which the EC approval is based, show that a single infusion of cilta-cel resulted in durable responses in a heavily pre-treated patient population. These results support the potential of cilta-cel in offering patients and physicians a valuable new treatment option.”

“At Janssen, we are building on nearly 20 years of commitment towards changing what a multiple myeloma diagnosis means for patients and are intent on addressing the high unmet needs that still remain,” said Edmond Chan, MBChB M.D. (Res), EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. “With today’s approval of cilta-cel in Europe, we are pleased to be delivering a new approach to the way in which healthcare professionals can tackle, and hopefully one day overcome, this complex disease.”

Conditional marketing authorisation (CMA) is the approval of a medicine that addresses unmet medical needs of patients based on less comprehensive data than normally required, where the benefit of immediate availability of the medicine outweighs the risk, and the applicant is able to provide comprehensive clinical data in the future.[4] This CMA was supported by the pivotal CARTITUDE-1 study, including patients who had received a median of six prior treatment regimens (range, 3–18), and had previously received an IMiD, PI, and an anti-CD38 monoclonal antibody.1 Findings presented at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting showed that at a median duration of 18 months follow-up (range, 1.5–30.5), a one-time treatment with cilta-cel resulted in deep and durable responses, with 98 percent (95 percent Confidence Interval [CI], 92.7–99.7) of patients with RRMM responding to therapy (98 percent overall response rate ORR).1,6 Notably, 80 percent of patients achieved a stringent complete response (sCR), a measure in which a physician is unable to observe any signs or symptoms of disease via imaging or other tests after treatment..

“The approval of Janssen’s first cell therapy is testament to our ongoing commitment to advance science and transform outcomes for those living with multiple myeloma,” said Sen Zhuang, M.D., Ph.D., Vice President, Oncology Clinical Research, Janssen Research & Development, LLC. “We are deeply thankful to the patients, their families, the nurses, and the teams of researchers and study centres who have participated in the clinical study of cilta-cel and made today’s approval possible.”

The longer-term efficacy and safety profile of cilta-cel is being assessed in the ongoing CARTITUDE-1 study. Two-year follow-up results presented most recently at the American Society of Hematology (ASH) 2021 Annual Meeting showed that 98 percent of patients treated with cilta-cel for RRMM responded to therapy (98 percent ORR [n=97]), and the majority of patients achieved sustained depth of response, with 83 percent achieving a sCR at the 22-month follow-up.[3]

About Ciltacabtagene Autoleucel (cilta-cel)
Cilta-cel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA.[1],[3] BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells.[6],[7] The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA.[1] Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.[8]

About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.[16] In multiple myeloma, cancerous plasma cells change and grow out of control.[7] In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,500 patients died.[17] While some patients with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms, which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels or kidney failure.[18]

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 & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.

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