FDA grants orphan drug tag to NuCana’s biliary tract cancer treatment

NuCana is on its way to open a Global Phase III study of Acelarin in combination with cisplatin for patients who are suffering from the disease in 2019

NuCana plc recently announced that it has received Orphan Drug Designation for Acelarin, an investigational drug, from the U.S. Food and Drug Administration (FDA) to treat Biliary Tract Cancer. Acelarin is a ProTide transformation of gemcitabine and a new chemical entity that NuCana has produced.

Founder and Chief Executive Officer, Hugh Griffith said that there are considerable unmet requirements for patients suffering from this type of cancer.  The company performed an Phase 1b study on the combination of Acelarin and cisplatin that showed nearly double response rate, as compared with the standard of care ( gemcitabine plus cisplatin), he mentioned.

Griffith stated that many patients achieved a significant reduction in the volume of their tumor along with further shrinkage of the tumor with time. The company believes that Acelarin is a potential example of a notable advancement in biliary tract cancer and they are on their way to open their Global Phase III study with the combination of cisplatin for patients who are suffering from the disease in 2019, Griffith added.

For the record, Orphan Drug Designation is given to drugs by the FDA that are defined for prevention, diagnosis or treatment of rare diseases or conditions which are affecting less than 200,000 people in America. It provides benefits and incentives like prescription drug user fee waiver and tax credits for clinical trial cost.

For the uninitiated, NuCana plc is a clinical-stage biopharmaceutical company, which is focusing on enhancing treatment outcomes for patients suffering from cancer by applying the company’s ProTide Technology. It supposedly helps in transforming largely prescribed nucleoside analogs, chemotherapy agents into safer and more effective medicines.

Biliary tract cancer is reportedly a type of cancer that starts in the bile duct system connecting to liver, gallbladder and small intestine carrying digestive fluid through the liver.

 

Source Credit: https://www.globenewswire.com/news-release/2019/06/12/1868106/0/en/NuCana-Receives-Orphan-Drug-Designation-from-the-U-S-Food-and-Drug-Administration-for-Acelarin-for-the-Treatment-of-Biliary-Tract-Cancer.html