Crystal Genomics Announces FDA Orphan Drug Designation for CG-745 in Pancreatic Cancer

On August 30, 2019, VCBeat (WeChat ID: vcbeat) learned from foreign media reports that CrystalGenomics (KOSDAQ: 083790) announced that the U.S. Food and Drug Administration (FDA) had granted orphan drug designation to CG-745. CG-745 is a clinical-stage histone deacetylase (HDAC) inhibitor for the treatment of various hematologic malignancies and solid tumors, including pancreatic cancer.

Dr. Joong Myung Cho, Chairman and CEO of CrystalGenomics, stated, “We are pleased that CG-7455 has received FDA Orphan Drug Designation. Our Phase II clinical trial in pancreatic cancer is currently ongoing, and the results to date have been favorable.”

CG-745 is currently being evaluated in a Phase II clinical trial in combination with gemcitabine and erlotinib to assess its safety and efficacy in patients with locally advanced unresectable or metastatic pancreatic cancer. Additionally, another Phase II clinical trial is underway to evaluate the safety and efficacy of CG-745 in patients with myelodysplastic syndromes (MDS) who have failed prior hypomethylating agent therapy.

“Orphan drugs,” also known as rare-disease drugs, are pharmaceutical products used for the prevention, treatment, and diagnosis of rare diseases. Due to the small patient population, limited market demand, and high research and development costs, few pharmaceutical companies focus on developing therapies for these conditions; hence, these medicines are vividly termed “orphan drugs.” Currently, China has a significant gap in the research and development of orphan drugs, with treatments for rare diseases relying largely on imports. As a result, many patients with rare diseases are left with no choice but to use expensive imported medications or face a complete lack of available treatments.

FDA Orphan Drug Designation is a status granted by the FDA to drugs that meet the criteria under the orphan drug policy. Upon receiving this designation, the drug becomes eligible for various incentives outlined in the U.S. Orphan Drug Act during its regulatory review and approval process in the United States.

Pancreatic cancer is a fatal disease and one of the leading causes of cancer-related mortality in the United States. The incidence of pancreatic cancer has increased over the past few decades, and it is considered the fourth leading cause of cancer deaths in the U.S., as the five-year survival rate for patients with this fatal disease is only 8.5%. Due to the poor response of pancreatic tumors to chemotherapy or radiotherapy alone, surgical resection remains the only known curative treatment for patients with localized disease. However, patients with advanced or recurrent forms of the disease do not benefit from surgery, and their overall survival rate is merely 11.5% due to the limited availability of effective therapeutic options for these patients.

CrystalGenomics is a biopharmaceutical company founded on protein structural chemistry, dedicated to the research, development, and commercialization of innovative drugs to address needs in the fields of inflammation, oncology, and infectious diseases. Headquartered in Seoul, South Korea, the company maintains a global development division in California, USA, and is listed on the KOSDAQ exchange (stock code: 083790).

(Compiled by Tian Shuhang)