On August 28, 2019 CrystalGenomics, Inc. (KOSDAQ: 083790) reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CG-745, a clinical stage histone deacetylase (HDAC) inhibitor being developed for several hematologic malignancies and solid tumors, for the treatment of patients with pancreatic cancer (Press release, CrystalGenomics, AUG 28, 2019, View Source;id=1821&page=1&num=126&nowpos=163&type=&sermun=&qu=&tb_name=eng_news&rt_page=/en/news/news.php [SID1234539162]).
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Dr. Joong Myung Cho, Chairman and Chief Executive Officer of CrystalGenomics, said, "We are pleased that CG-745 has received Orphan Drug Designation from the FDA for the treatment of pancreatic cancer. Currently our Phase II pancreatic cancer trial is still ongoing, and the results look promising thus far."
CG-745 is currently being studied in a Phase II clinical trial in combination with gemcitabine and erlotinib to evaluate its safety and efficacy for locally advanced unresectable, or metastatic pancreatic cancer. Additionally, there is another ongoing Phase II clinical trial to evaluate the safety and efficacy of CG-745 in patients with Myelodysplastic Syndrome (MDS) who have failed hypomethylating agents.
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides incentives for sponsors to develop drugs and biologics for rare diseases. These incentives may include a partial tax credit for certain clinical trial expenditures, the waiver of certain FDA user fees, and if approved, potential eligibility for seven years of orphan drug marketing exclusivity, which is independent from intellectual property protection.
About Pancreatic Cancer
Pancreatic cancer is a deadly disease and one of the leading causes of cancer deaths in the US. The incidence of pancreatic cancer has increased over the past several decades and is considered the fourth leading cause of cancer deaths in the US as patients with this deadly disease have a 5-year survival rate of 8.5%. As pancreatic cancer tumors respond poorly to standalone chemotherapy and radiation, surgical resection is the only known curative treatment for patients with disease localized to the pancreas. However, patients with advanced or recurrent forms of the disease are not able to benefit from surgery, leaving these patients with limited effective treatment options and an overall survival of only up to 11.5%. Unfortunately, only 10% of pancreatic cancer patients have localized disease, leaving a large unmet medical need for treatment in 90% of the pancreatic cancer patient population.