The U.S. Food and Drug Administration has granted orphan drug designation to CPI-613 for the treatment of myelodysplastic syndrome. The drug is a first-in-class mitochondrial metabolism inhibitor that targets pyruvate dehydrogenase (PDH) and alpha ketoglutarate dehydrogenase (KGDH) enzymes to cause tumor cell death.
Orphan drug designation—to encourage development of drugs in the diagnosis, prevention, or treatment of a medical condition affecting fewer than 200,000 people in the U.S.—grants a product market exclusivity for a seven-year period if the sponsor complies with certain FDA specifications, as well as tax credits and prescription drug user fee waivers. The designation does not, though, shorten the duration of the regulatory review and approval process.
Approximately 12,000 patients are diagnosed with MDS each year, according to estimates from the American Cancer Society.
CPI-613 is currently being evaluated in Phase I and I/II, and Phase II trials in hematologic malignancies and solid tumors. The results from the Phase I single agent trial in patients with advanced hematologic malignancies were presented at this year's American Society of Clinical Oncology Annual Meeting (Abstract 2516).
The drug is marketed by Cornerstone Pharmaceuticals.