The FDA has granted Orphan Drug Designation to CX-4945 for the treatment of cholangiocarcinoma.
CX-4945 is a novel small molecule drug that inhibits protein kinase CK2, which plays an important role in the DNA damage repair mechanisms of cancer cells.
The study drug has demonstrated favorable safety, pharmacokinetic, and pharmacodynamic characteristics in the treatment of advanced cholangiocarcinoma, a disease for which there are limited effective therapies. This cancer is difficult to detect in its early stages, and the survival rate at 5 years is only around 20 percent.
Orphan Drug Designation is granted by the FDA to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the U.S. This Orphan Drug Designation will expedite the development of CX-4945 in the treatment of cholangiocarcinoma.