In a news release from late May 2022, pharmaceutical company SteroTherapeutics LLC, known for developing orphan drugs for rare conditions, shared that its novel therapy ST-003 received Orphan Drug designation for primary sclerosing cholangitis (PSC). The FDA grants this designation to drugs or biologics intended to treat, prevent, or diagnose rare diseases. These are conditions affecting fewer than 200,000 Americans. As a benefit, SteroTherapeutics also earns a variety of incentives such as fee waivers, tax credits, increased regulatory assistance, and 7 years of market exclusivity upon drug approval.
What is ST-003?
ST-003 is a novel galanin inhibitor. The drug is designed to stop excess galanin from accumulating, which causes issues such as fibrotic scarring and cholangiocyte proliferation. To learn more about ST-003 and how it works, as well as to understand more about galanin and its links to disease, take a look at this helpful guide on the SteroTherapeutics website.
Primary Sclerosing Cholangitis (PSC): An Overview
An estimated 70% of individuals who have primary sclerosing cholangitis (PSC), a chronic disease which causes progressive bile duct damage, are male. The cause of PSC is unknown. Some researchers hypothesize that PSC results from viral or bacterial infections, while others believe that the cause could be autoimmune, due to toxin exposure, or related to inflammatory bowel disease (IBD). Regardless, PSC develops when bile ducts undergo scarring and inflammation, which prevents and blocks bile from being transported from the liver to the gallbladder and small intestine. As bile accumulates, it causes damage, scarring, and even liver failure. Right now, there are no cures for PSC. However, in 80% of cases, a liver transplant can increase the survival rate to 2+ years.
When PSC first develops, patients may experience symptoms such as abdominal discomfort, severe itching, and fatigue. However, as the disease progresses, symptoms can include:
- Fever
- An enlarged liver and spleen
- Osteoporosis
- Jaundice (yellowing of the skin, eyes, and mucus membranes)
- Bile duct infection
- Upper-right abdominal pain
- Liver failure
