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                Resources for Information | Approved Drugs

Resources for Information | Approved Drugs

                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
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          Resources for Information | Approved Drugs
          FDA D.I.S.C.O. Burst Edition: FDA approval of CARVYKTI (ciltacabtagene autoleucel) for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibodyResources for Information | Approved Drugs FDA D.I.S.C.O. Burst Edition: FDA approval of CARVYKTI (ciltacabtagene autoleucel) for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody
  
  	
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Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide a quick update on a recent FDA cancer therapeutic approval.

On February 28, 2022, the FDA approved ciltacabtagene autoleucel (brand name CARVYKTI) for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Ciltacabtagene autoleucel is a B-cell maturation antigen-directed genetically modified autologous chimeric antigen receptor T-cell therapy. Each dose is customized using a patient’s own T-cells, which are collected and genetically modified, and infused back into the patient.

Safety and efficacy were evaluated in CARTITUDE-1, an open label, multicenter clinical trial evaluating ciltacabtagene autoleucel in 97 patients with relapsed or refractory multiple myeloma who received at least three prior lines of therapy which included a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody and who had disease progression on or after the last chemotherapy regimen. Patients received ciltacabtagene autoleucel within the range of 0.5 1.0×106 CAR-positive viable T cells per kg body weight. Efficacy was established based on overall response rate, and duration of response, as evaluated by an Independent Review committee using the International Myeloma Working Group Uniform Response Criteria for Multiple Myeloma. The overall response rate was 97.9%. Among the 95 patients who responded, the median duration of response was 21.8 months with a median duration of follow up of 18 months.

The CARVYKTI label carries a boxed warning for cytokine release syndrome, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, Immune Effector Cell-Associated Neurotoxicity Syndrome, Parkinsonism and Guillain-Barré syndrome and their associated complications, and prolonged and/or recurrent cytopenia, all of which can be fatal or life-threatening.

CARVYKTI is approved with a risk evaluation and mitigation strategy requiring that hospitals and their associated clinics that dispense the therapy must be specially certified to recognize and manage cytokine release syndrome and nervous system toxicities. To evaluate long-term safety, the FDA is requiring the manufacturer to conduct a post-marketing observational study involving patients treated with ciltacabtagene autoleucel.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

Full prescribing information for this approval can be found on the web at www.fda.gov, with key word search “Approved Cellular and Gene Therapy Products”.

Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.

Follow the Division of Drug Information on Twitter @FDA_Drug_Info and the Oncology Center of Excellence @FDAOncology. Send your feedback via email to FDAOncology@fda.hhs.gov. And thanks for tuning in to the D.I.S.C.O. Burst Edition.

                                                                                  Content current as of:
      03/30/2022Resources for Information | Approved Drugs

          
                        Oncology (Cancer) / Hematologic Malignancies Approval Notifications
          Ongoing | Cancer Accelerated Approvals
          Verified Clinical Benefit | Cancer Accelerated Approvals
          Withdrawn | Cancer Accelerated Approvals
          Other | Cancer Accelerated Approvals


                        Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) Short Description
 
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