European Commission grants conditional approval of CARVYKTI® (Ciltacabtagene Autoleucel), Janssen’s first cell therapy for the treatment of patients with relapsed and refractory multiple myeloma.

Beerse/Breda, 26 May 2022 – 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 adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody, and have demonstrated disease progression on the last therapy.[1] 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.[2]

Cilta-cel is a chimeric antigen receptor T-cell (CAR-T) therapy featuring two B-cell maturation antigen (BCMA)-targeting single domain antibodies.[3],[4],[5] CAR-T therapy is a highly personalised technology where a patient’s own T-cells are re-programmed to target and kill cancer cells – and is administered as a single infusion.[3],[6]

The longer-term efficacy and safety profile of cilta-cel is being assessed in the ongoing CARTITUDE-1 trial. The results of the 28-month median follow-up presented at the American Society of Clinical Oncology (ASCO) 2022 Annual Meeting showed that 98% of RRMM patients treated with cilta-cel responded to this therapy (98% overall response rate [ORR], n=97). The majority of these patients achieved a sustained deep response, of which 83% achieved a stringent complete response (sCR).[3] The results of the 12 month median follow-up have already been published in the medical journal “The Lancet”.[4]

The most common adverse reactions (≥ 20%) of cilta-cel were neutropenia (91%), Cytokine Release Syndrome (CRS) (88%), pyrexia (88%), thrombocytopenia (73%), anemia (72%), leukopenia (54%), lymphopenia (45%), musculoskeletal pain (43%), hypotension (41%), fatigue (40%), transaminases increase (37%), upper respiratory tract infection (32%), diarrhea (28%), hypocalcaemia (27%), hypophosphatemia (26%), nausea (26%), headache (25%), cough (25%), tachycardia (23%), chills (23%), encephalopathy (22%), decreased appetite (22%), edema (22%) and hypokalaemia (20%).[1]

As a highly personalized medicine where a patient’s own T-cells are reprogrammed to target and kill cancer cells,[4] administration of CAR-T therapy requires extensive training, preparation, and certification to ensure the highest quality product and experience for patients. In the coming period, Janssen will strive for a phased activation of a European network of certified treatment centers.

About Ciltacabtagene Autoleucel (cilta-cel)

In addition to the Breakthrough Therapy Designation in the United States (US) awarded in December 2019,[7] cilta-cel received a PRIority MEdicines (PRiME) - designation from the European Commission (EC) in April 2019[8] and a Breakthrough Therapy Designation in China in August 2020.[9] Janssen also received orphan drug designation for cilta-cel[10] from the EC in February 2020 and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In May 2022, the Committee for Orphan Medicinal Products recommended by consensus that orphan drug designation be maintained for cilta-cel, based on clinical data demonstrating improved and sustained complete response rates after treatment.[11] Cilta-cel received approval from the U.S. Food and Drug Administration (FDA) in February 2022.[12]

CARTITUDE-1 (NCT03548207)[5] is an ongoing Phase 1b/2, open-label, multicenter study evaluating cilta-cel for the treatment of patients with RRMM who previously received a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and received an anti-CD38 antibody and who showed disease progression on or after the last regimen. All patients in the study had received a median of six prior treatment regimens (range, 3-18).[13] Of the 97 patients enrolled in the study, 99% were refractory to the last line of treatment and 88% were three-fold refractory, meaning that their cancer did not or no longer responded to an IMiD, a PI and an anti-CD38 antibody.[13]

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. When damaged, these plasma cells change and grow out of control.[14] In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,500 patients died.[15] 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.[16]

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.

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 materialise, actual results could vary materially from the expectations and projections of Janssen Pharmaceutica NV, Janssen Biotech, Inc., Janssen-Cilag Limited and Janssen Research & Development, LLC, and, and 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 2, 2022, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other 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.

This medicine is subject to additional monitoring.

* CARVYKTI® was developed in collaboration with Legend Biotech. See press release for more information: 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

CP-314791 - July 2022