Experimental Treatment for Pancreatic Cancer has Earned Orphan Drug Designation

According to a story from BioSpace, the biopharmaceutical company CrystalGenomics, Inc. recently announced that the US Food and Drug Administration (FDA) has granted the company’s investigational drug candidate CG-745 Orphan Drug designation. This experimental therapy has earned the designation for treating pancreatic cancer and is also being developed to treat a variety of solid tumor and blood cancers. The drug is classified as a histone deacetylase inhibitor.

About Pancreatic Cancer

Pancreatic cancer is one of the most dangerous forms of cancer. The disease affects the pancreas, which is a glandular organ that is situated behind the stomach. Part of the reason that pancreatic cancer is so dangerous is that it rarely produces noticeable symptoms until it has reached an advanced stage and begun to spread. However, even when detected earlier, it is difficult to treat effectively. Risk factors for pancreatic cancer include being male, old age, African-American ancestry, family history, smoking, obesity, diabetes, chronic pancreatitis, and a diet heavy in red meat, processed meat, or meat cooked at very high temperatures. Symptoms include depression, upper abdominal pain, jaundice, diabetes, constipation, weight loss, and appetite loss. Treatment approaches for this cancer include surgery, chemotherapy, and radiation therapy. Even with heavy treatment, pancreatic cancer almost always returns. The five year survival rate is just five percent. To learn more about pancreatic cancer, click here.

Orphan Drug Designation

Orphan Drug designation is intended for therapies that are being developed to treat diseases that are considered rare. This is defined as any disease that affects less than 200,000 people in the US. To qualify for this designation, a treatment must display potential advantages in safety or efficacy in comparison to treatments that are currently available. Alternatively, it must fulfill a currently unmet medical need. Orphan Drug designation confers several benefits to the recipient company, including tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the FDA approves the drug.


CG-745 is currently undergoing testing in a pair of phase 2 clinical trials. One trial is testing the drug in combination with the medications erlotinib and gemcitabine for metastatic or locally advanced pancreatic cancer that is not eligible for surgical removal. The other trial is testing the investigational drug in myelodysplastic syndromes patients that have failed to respond to hypomethylating agents. 

Follow us