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FDA Grants Orphan Drug Designation to CPI-613 for Treatment of Burkitt Lymphoma

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The U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to CPI-613 for the treatment of Burkitt lymphoma.

Burkitt lymphoma is a highly aggressive hematologic B-cell malignancy classically characterized by the overexpression of c-Myc. Due to the rapid proliferation rate of these tumors, the mainstay of treatment includes aggressive chemotherapy and immunotherapy. As per the National Comprehensive Cancer Network® (NCCN®) Guidelines, there is no definitive second-line therapy for Burkitt lymphoma (NCCN Guidelines®, Version 4.2018). There is a clear unmet medical need for additional treatment options for these patients.

CPI-613 is a novel lipoic acid analog with an anticancer activity that inhibits multiple enzyme targets within the tricarboxylic acid cycle. In a phase I clinical study of CPI-613 in patients with advanced hematologic malignancies (CL-CPI-613-009), a 19-year-old woman with relapsed Burkitt lymphoma was treated with CPI-613 monotherapy. After second relapse to the prior therapy, the patient was enrolled in CL-CPI-613-009 and received CPI-613 monotherapy (2,940 mg/m2).

The patient received 17 total cycles of therapy over 51 weeks and achieved and maintained radiographic partial response after the third cycle. The treatment was well tolerated. After 17 cycles, the patient decided to discontinue treatment and pursue a surgical resection of residual tumor. The pathology of the surgical specimen revealed Burkitt lymphoma with extensive necrosis. Clinical follow-up on the patient indicated no evidence of disease more than 36 months later.

A new phase II study of CPI-613 will look at the response rate of patients with relapsed or refractory Burkitt Lymphoma/leukemia and high-grade B-cell lymphoma with rearrangements of MYC and BCL2 and/or BCL6 (DHL/THL) treated with CPI-613.

Ariela Noy, MD, a medical oncologist specializing in lymphoma and AIDS-associated cancers at Memorial Sloan Kettering Cancer Center and Chair of the Lymphoma Working Group for the AIDS Malignancy Consortium, is the principal investigator of the phase II study.

Dr. Noy commented, “We look forward to launching this study with CPI-613 for patients with relapsed/refractory Burkitt lymphoma who do not have any viable treatment options. This could be a potentially life-saving therapy.”

The content in this post has not been reviewed by the American Society of Clinical Oncology, Inc. (ASCO®) and does not necessarily reflect the ideas and opinions of ASCO®.


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