CPI-613 Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma

CPI-613 is an altered energy metabolism directed (AEMD) drug candidate
CPI-613 is an altered energy metabolism directed (AEMD) drug candidate

Rafael Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug Designation to CPI-613 for the treatment of peripheral T-cell lymphoma (PTCL)

CPI-613 is an altered energy metabolism directed (AEMD) drug candidate being studied in combination with bendamustine in patients with relapsed or refractory T-cell lymphoma. The novel lipoic acid analogue works by inhibiting multiple enzyme targets within the tricarboxylic acid cycle. 

Currently, 10 patients received a dose of the study drug of which 7 were evaluated for efficacy. The primary objective is to determine the maximum tolerated dose (MTD) and safety profile when given with bendamustine.  Interim data showed CPI-613 plus bendamustine showed favorable efficacy with 86% overall response rate (43% complete response and 43% partial response). 

Related Articles

“This study of CPI-613 in combination with bendamustine in patients with relapsed or refractory T-cell lymphoma showed a good safety profile and encouraging efficacy. The orphan drug designation in this case provides hope to the poor-risk patient population, and inspires us to continue our tireless effort to bring these patients a legitimate treatment option," stated principal investigator Zanetta Lamar, MD, from Wake Forest Baptist. 

This first-in-class drug was previously granted Orphan Drug designation for the treatment of pancreatic cancer, acute myeloid leukemia, Burkitt lymphoma, and myelodysplastic syndromes.

For more information visit RafaelPharma.com.