Experimental Treatment for Primary Biliary Cholangitis Earns Orphan Drug Status in the US and EU

According to a story from Markets Insider, the biopharmaceutical company GENFIT recently announced that both the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) have awarded the company’s experimental drug elafibranor Orphan Drug designation. The designation was given to the drug as a treatment for the rare disease primary biliary cholangitis, a liver disease that affects the bile ducts. GENFIT is focused on the development of innovative new therapies for metabolic diseases and liver related diseases.

About Primary Biliary Cholangitis (PBC)

Primary biliary cholangitis, less commonly referred to as primary biliary cirrhosis, is an autoimmune disease the affects the liver. It is most characterized by progressive damage to the bile ducts, which over time allows bile and other toxic substances to build up in the liver. The disease can ultimately progress to liver scarring and cirrhosis. The precise cause of the disease remains unknown; a genetic predisposition, perhaps involving the POGLUT1 gene, is a risk factor, as is the presence of another autoimmune disease. Women are also much more likely to get primary biliary cholangitis. Symptoms include reduced bone density, skin lesions, fatigue (sometimes severe), jaundice, abdominal swelling, hepatic encephalopathy, and enlarged spleen. Treatment for the disease may include the drug Ursodiol, vitamin supplementation, and liver transplant in severe cases. Some patients experience slowed disease progression to the extent that their lifespan and quality of life are not significantly affected. To learn more about primary biliary cholangitis, click here.

Orphan Drug Designation in the EU and US

Orphan Drug designation is intended for therapies meant to treat diseases that are considered rare. This is defined as any disease that affects less than 200,000 people in the US or 5 in every 10,000 people in the EU. To qualify, the drug must satisfy an unmet medical need or display advantages in either effectiveness or safety over currently available treatments. In the US, Orphan Drug designation allows incentives such as the waiving of certain fees, tax breaks, and a seven year period of market exclusivity if the drug is approved. In the EU, incentives include protocol assistance from the EMA, fee reductions, access to the centralised authorisation procedure, and a ten year period of market exclusivity if approved.


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