GNS561 Earns Orphan Drug Designation for Cholangiocarcinoma


According to an article from Cancer Network, the U.S. Food and Drug Administration (FDA) recently granted Orphan Drug designation to second-line GNS561 (ezurpimtrostrat) for cholangiocarcinoma (bile duct cancer). 

In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions (rare = affecting fewer than 200,000 people in the country). Drug developers earn incentives such as fee waivers, tax credits, and seven years of market exclusivity upon approval. 

This designation was granted to GNS561, a new autophagy inhibitor, following the drug’s evaluation in a Phase 1/2a clinical study. People with intrahepatic cholangiocarcinoma, as well as hepatocellular carcinoma (HCC), enrolled in the study. Altogether, 50 participants enrolled. During the trial, researchers explored escalating doses of GNS561 to find the dose-limiting toxicity. 

More research – in the form of a Phase 2 study – is planned for the future. 

What is Cholangiocarcinoma?

Also known as bile duct cancer, cholangiocarcinoma is a rare cancer that forms in the bile ducts, which carry bile from the gallbladder to the small intestine. Based on which parts of the bile ducts are affected, cholangiocarcinoma can be divided into the categories of hilar, distal, or intrahepatic. 

Risk factors include having primary sclerosing cholangitis or chronic liver disease, congenital bile duct problems, a liver parasite, smoking cigarettes, having diabetes, obesity, biliary cysts, having certain inherited conditions, or being older in age.

Unfortunately, cholangiocarcinoma can be difficult to treat. This is because the cancer can be asymptomatic in early stages and is often not diagnosed until it is advanced. When symptoms appear, they can (but do not always) include:

  • Appetite loss
  • Dark urine and pale stools
  • Nausea and vomiting
  • Abdominal pain on the right side (below the ribs)
  • Fever
  • Chills and night sweats
  • Pruritus (intensely itchy skin)
  • Unintended weight loss
  • Jaundice (yellowing of the skin, eyes, and mucous membranes) 

Unfortunately, cholangiocarcinoma can be difficult to treat, lending to a poor prognosis. This is why additional options, beyond the current first-line treatment options, are urgently needed.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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